32 Burst results for "Sickle Cell Disease"

Nature Makes GMO Fish

Science Facts & Fallacies

02:11 min | Last month

Nature Makes GMO Fish

"Skeptical of aqua bounties fast-growing salmon while nature makes genetically modified fish to next up. Why the effort to find a quote biological basis for being transgender is misguided and harmful and finally cure for sickle cell diseases inches closer with the launch of a major gene. Therapy trial very exciting stuff. But first up kevin. Let's talk about Natural gm fish apparently. Yeah this is really cool and this is a report that came out in. Cbc news which is canadian broadcasting company usually not writing very favorable things about genetic engineering because this isn't genetic engineering unless it's genetic engineering that nature did so the story goes back to a couple of researchers at queens university. A lorry graham and peter davies found this evidence a while ago now that there was this antifreeze gene that helped the rainbow smelt live through freezing temperatures so super cooling fish and some other organisms can endure temperatures below freezing because of a mechanisms that keep their cells from forming ice crystals. What interesting stuff going on there and if you think about it. These are cold-blooded so when it gets below freezing you have the risk of freezing so years ago. Probably a decade and a half. I guess they found this evidence that there was A this gene that that didn't seem to make a whole lot of sense that that it that it looked like a in so we go over now to herring and smelt How was this hearing. Gene ending up and smelt the last time that they were related was by extremely distant ancestor. They said by the article article here. Two hundred and fifty million years ago so that was like the carbon difference period so these two individual lineages of fish split off pretty early a back before dinosaurs or you know and so this really cool so well same time as early dinosaurs.

Sickle Cell Diseases Cbc News Peter Davies Queens University GM Kevin Graham
Michael Friend Advocates For Diversity In CRISPR

CRISPR Cuts

04:34 min | 2 months ago

Michael Friend Advocates For Diversity In CRISPR

"Can you talk about what that role endangered so as a minority being part of minority coalition. How does that actually would say you're planning. Events are one out of the types of responsibilities. you're kind of involved in shore. What for chris. Carr my role on is on of the committee and really is to help develop strong. And broad foot trenton. Targeted monastic communities by by really joe trying to facilitate interactions with community based organizations to include aftermarket universities. Darkly restore cabrera colleges. Hvac us that had a very long standing history of mistrust to some extent. And so i kind of summit up just to really increase bernardi representation in these conversations around christopher and engagement with a very strong focus on making sure we have diverse forces some of our listeners. Who may not know exactly. What could a split on this. Can you just in a few lines. Dealers about guzman on absolutely or christopher is is an organization that really focused on the compensation crispin and they do it from the perspective where they're not be. The four or guest technology did not pro con but to really have conversations as it relates to this technology in house being utilized many ways and the benefits and risks down a bowl and started out by uc berkeley in conversations. There which later a year after moved to boston and she'll hope these. These conversations are kind of women around the country at really sparing Lotta interests around. Christopher and i can say that they've been very diverse voices after just faded indiscretions discussions. That's a very interesting role in vegas to learn more about your experiences by doing this. Just one question before that. How did you get into you. Know the crisper fields specifically was your background and christopher. How did that come to be that. You are involved in organized crisper con is. It's all started. President obama launch of the precision medicine issue which was pm. I in two thousand fifteen. I was invited to be a part of that much and that pm. I initiative is a multi year multi million on effort that was developed to keach around creating a diverse cohort to trutv by disease treatment for all but that program is currently known as all of us program and so from that launch. I started the minority coalition for position mets. So you're speaking under the bit of audio experiences. As an event organizer being part of that rising committee on crisper con- you had mentioned that when pulling together some event it was really impossible for you to find black researchers in the area. So could you elaborate more on the garden. Status stakes are the neck of diversity that you are seeing in the field. Well just to be honest. You know this is that i think at this point is kinda shameful in subsets. Your article article that you've written highlighting some of top companies in fear. Know as you look at even these compromises need couples this leading the work around crisper. You can see most senior today. Shes clearly the lack of diversity that so evident in these companies. And i think what is sometimes hurtful. Is that in this crisper in some ways to successive crisper. Really an johnny outcome A black disease sickle cell disease which is driving an has driven a lot of interest at of course financial investments. And so i would say at this point. It's not looking very good chance of diversity

Christopher Uc Berkeley Bernardi Trenton Cabrera Carr Guzman Crispin Keach Minority Coalition For Positio Chris JOE Boston President Obama Vegas Sickle Cell Disease Johnny
Gene-editing treatment shows promise for sickle cell disease

Morning News with Hal Jay & Brian Estridge

03:09 min | 5 months ago

Gene-editing treatment shows promise for sickle cell disease

"Preliminary data out there that they're calling nothing short of great. It's new data on crisper treatments for blood diseases, Okay? This crisper C l c R i s p R is gene editing therapy? Okay, and this is specifically dealing with blood disorders. That are inherited things like sickle cell. Okay, okay, And these folks who have been treated with this gene editing therapy have shown consistent and sustained response with manageable side effects. The these two crisper therapeutics is the name of the company. They in a Vertex Pharmaceuticals are jointly developing a one time gene editing treatment. They call it C t x 001. And I know they were kind of getting in the weeds here. But here's the deal after giving the therapy by infusion, all the patients in both studies. Have been free from symptoms of the disease is and have not needed blood transfusions. Wow, that's amazing, all of them all of them in free of the symptoms Now, do they have an in home model that you could take up with? It's not there yet course But this This is huge, though. Um, they're also going to be some clinical trial results on the sickle cell gene therapy from another group called Bluebird Bio. S So you got a couple of gene therapies that are taking a look at this sickle cell anemia. You also have beta thalassemia is another one. I'm sure I'm saying that wrong Well, last Mia, but it says Hey, We've made a real pivot and what we've been waiting for. And now we're finding the ability to offer patients long term disease control using genetics and genetic therapy. I've heard some reports on this and one of the woman women that got the therapy. Said It was night and day. The chicken function. I mean, she and I didn't realize how painful Oh, yeah. Sickle cell is and yeah, she said she has a life again. Yeah, it was like Almost immediate Like when they got the confusion. That is awesome. Yeah. Inwood sickle cell with the sickle red cells become clogged and blood vessels and then you get extreme pain that can also lead to like organ damage, stroke, early death things like that. Uh, And so, I mean, this could be huge. How That if they can figure out a way to just edit the gene that's causing sickle cell and they do it with a one time infusion and then your transfusion free. So this this this This could game changer for all different kinds of cheeses. How about this out? The average sickle cell patient right now has to have 15 blood transfusions a year. You gotta be kidding. No, I had no idea and this would eliminate that. That is so bad for the one time infusion.

Vertex Pharmaceuticals Bluebird Bio Thalassemia Inwood Sickle Anemia Sickle Confusion Stroke
Minnesota adds 2 more qualifying conditions for medical marijuana

Buck Sexton

00:18 sec | 5 months ago

Minnesota adds 2 more qualifying conditions for medical marijuana

"Is adding sickle cell disease vocal and motor TIC disorder patients to its medical marijuana program. The state says the new qualifying conditions will take effect in August, joining the 15 other conditions that have been approved in past years. The Department of Health says a petition for anxiety was rejected this year, but they plan to revisit the application next year

Sickle Cell Disease Department Of Health
Berlin patient: First person cured of HIV, Timothy Ray Brown, dies

Morning News with Manda Factor and Gregg Hersholt

01:41 min | 8 months ago

Berlin patient: First person cured of HIV, Timothy Ray Brown, dies

"Cured of HIV passed away this week, and we're learning that his influence on local research here lives on comas. Denise Whitaker has that story. Timothy Ray Brown was a Seattle native but living in Germany when he was cured of HIV through a bone marrow transplant. I interviewed him in 2013 after he moved back to Seattle from Germany, where he received that transplant and became known as the Berlin Patient. Brown passed away this week at age 54 still free of HIV, but his cancer had returned. His case continues to inspire researchers of the Fred Hutchinson Cancer Research Center, the first pioneers of bone marrow transplants back in the 19 sixties. And that research continues. Today. Dr. Hans Peter Quim tells me that what started with Brown in Berlin more than 20 years ago, continues to influence the research into many other diseases. Today, we knew by then, already that we could, in fact, modify a patient's own cells to making resistant to HIV, that really no launch this of HIV effort at that time, and my lab Ridgeway and microbe has really worked on are now using the patient's own cells. To make the patient's own cells resistant to HIV team just received a new grand from the National Institutes of Health to continue his research of the hutch. He's essentially kind of gene editing kind of taking jeans apart and modifying them, putting them back into patients. Researchers at the Hutch have expressed their sadness at Brown's death, but say his story really continues to give them and others suffering from HIV. Sickle cell disease and other blood diseases. But there is hope for more to be cured. Come on news time.

HIV Timothy Ray Brown Fred Hutchinson Cancer Researc Berlin Denise Whitaker Germany Dr. Hans Peter Quim National Institutes Of Health Seattle
Freedom Summer: Barbara Lee

The Brown Girls Guide to Politics

07:06 min | 9 months ago

Freedom Summer: Barbara Lee

"In June nineteen sixty four freedom summer also known as the Mississippi Summer Project was a volunteer campaign across America to attempt to register as many black American voters as possible in Mississippi. News coverage of freedom summer shed a light on the white supremacy and police brutality that black Americans face. We. Don't Tuesday night the finding of three bodies in graves at the site of a damn near Philadelphia Mississippi where three civil rights workers disappeared six weeks ago. Over the past few weeks we have been experiencing another freedom summer. Minnesota are saying to people in New York two people in California to people in Memphis to people all across this nation enough is enough cell phone videos and social media are once again providing glaring spotlight on the inequities and injustice that are woven into the fabric of American society. In this special season of the browns to politics, we are diving into the past in how is impacting our present and future. For protests to political campaigns and youth involvement change is in the air and the fight for liberation continues. We'll be hearing from some of the Black Women at the forefront at today's movement who are fighting for change in making history to ensure that we have justice for all. Her name was even floated as a potential. VP. Pick for Democratic presidential nominee Joe Biden's. It is no surprise that would ever congress is debating issues of equity and justice. Congress will lease voice is one of the strongest and most prominent today we talk about her work as a college student, a member of the Black Panther Party and what Congress is, do we to fight systems of oppression to reshape reimagined our political world? Congresswoman Barbara Lee thank you so much for joining us and happy belated birthday. Breaking very good happy with you. I'm really excited to talk to you today and for our listeners, the congresswoman is such a legend and all of her work that she has done in. Congress over the years especially for Black Brown and indigenous communities by I have to ask you this question because it's something that I just wanted to talk to you about for so long is. You were a part of the Black Panthers. What was it like being? Black Panther I actually was not a member of the Black Panther Party I was what they call the community worker community workers had a lot of responsibilities as the Black Panther. Party. Members and remember the Black Panther Party began as a result of police Gupta brutality and the African American community. I mean. They stood down the police because things, police, murders, police Retali- as we know now were occurring then and they were the first organization that really took the police on, and so it was out of that that the Black Panther party formed, there's the Bible programs because it was not only an organization that address police brutality, but it was an organization that addresses chemic-. Racism and poverty. and. So what I did, and which was really phenomenal work and I was a single mother on public assistance with two little boys. I helped sell newspapers like math a newspaper on street corners I actually participated in the breakfast program for children who didn't have whose parents didn't have enough money to buy food and that's actually the breakfast programs from the federal government. Actually. Started as a result of the of the models that the Black Panther party you. I also really worked with you. He knew then did the research on his book Revolutionary Suicide. It was really phenomenal project I got to know Huey Newton Bobby Seale, Elaine Brown, Erica Huggins Joan Kelly, who just passed away and many of the leadership of the black. Panther party because community worker and student I was very involved in a lot of the work with party members. I actually brought Shirley Chisholm got involved in politics through the first presidential the first. Time. A black woman ran for president and that was sure children who was the first African American woman elected to Congress and so the Black Student Union president I invited her to come to milk college where I was attending and I got involved in her campaign by herb insisting that I register the vote and I had a class go because I didn't WanNa work in any of those campaigns. Well, bottom line is working her campaign and got the Black Panther party really involved in voter registration efforts. I. Was the one that went and asked Huey Noonan Bobby Seale to consider becoming politically active around early Chisholm campaign and they did. So I worked on all phases of the black. Panther. Party and all the different divisions I actually bag groceries. You know the panthers had a whole ten point program which again, the Free Breakfast program for the kids They started the Community Health Center Movement by instituting the George Jackson free medical clinic they did sickle cell tests. In fact, there was the Black Panther party that raises awareness about sickle cell disease as a as a disproportionate impact African Americans Fast Board Twenty Twenty people in the African American community and Black and Brown news still struggling disproportionately as it related to food security food desert healthcare disparities, unequal education. I. Helped. Start. Actually I wrote the first proposals for the Black Panther Party community learning center. They establish a Black Panther party school and so I was very instrumental in working on that project. So I did a lot of work with the Black Panther Party and I can just speak to how phenomenal they were and how necessary they were and how we should as we move forward. You know there's this Symbol in a gun and Andy. In government in Ghana called and Copeland. If the bird beautiful bird looking back holding an egg in her mouth and like in order to move forward in order to blackboard and you have to look back, we have to know our history we know where we've been and we have to build upon that so that we can move forward it. Now a wonderful young people in the Movement for Black, flags, or dreamers all the movements that are taking place are a continuation of what I see as the civil rights movement of of today, as well as what Black Panther Party actually started as it relates to stand down and and thing that that policing in our community. chain stop disproportionate killing black, and Brown people

Black Panther Party Black Panther Party School Panther Party Congress Black Women African American Community Black Student Union Congresswoman Barbara Lee Mississippi Huey Noonan Bobby Seale Philadelphia Mississippi Joe Biden Minnesota Browns Shirley Chisholm Memphis Ghana Panthers New York
"sickle cell disease" Discussed on This Podcast Will Kill You

This Podcast Will Kill You

05:28 min | 9 months ago

"sickle cell disease" Discussed on This Podcast Will Kill You

"Beta thou- me or these other blood disorders. That's GONNA be a lot of money, and then in theory perhaps paying half a million dollars once is actually going to be cheaper and then the long term cost. If. You can't afford that upfront. It's it's a moot point and I think right now we're kind of trying to figure out if this is something that's going to be covered by insurance companies I think it's an issue in America. That kind of broader than the science but. We've been trying to think if there are scientific solutions. So hopefully, someone will figure out and social solutions to health care A. But in the meantime we're we're trying to figure out if there are ways that we can change the way we do the science that will actually change the outcome when when things are priced eventually. So that's that's one thing that we're working on that I'm kind of hopeful about one of the big issues with sickle cell disease that's going to make this so expensive is that we're doing. All of this gene editing I'm talking about inpatient cells that we've taken out of patient. So we're not putting a shot in someone's arm or giving them a pill were taking their cells, extracting cells from their bone marrow, editing them in the lab and putting them back into the patient's body, and that's really complicated and expensive requires people with a lot of expertise to handle the cells and it's it just jack up the price by a lot and there's also this requirement in a lot of these cases for using virus to deliver. The the crisper tools and manufacturing this virus is really expensive and difficult as well. So there are a lot of steps and compounding steps potentially add cost. So we've been trying to think, are there ways to do this in Vivo? So instead of having to take cells out and put them back, can we just do the fixed directly in a patient's body? So I think there are potential scientific solutions to some of these problems, but they're really really hard problems and they'll take a lot of investment you said you've talked. In. This episode about kind of historic marginalization of people with sickle cell and the way there's you know racism and medicine manifest. In this disease I think one of the promising things that's been happening lately is one this kind of reckoning of -mongst the white scientific community and others about how lack communities have been affected by the practice of science and government funding and medicine, and all of that and to we were recently told that the NIH and Gates Foundation are now investing one hundred, million dollars towards doing in. Vivo therapies. Or potentially other therapies but particularly in Vivo therapies using genome editing for sickle cell so that that's the huge investments of money that I think could make a really big difference in how we're able to treat disease him actually making it an affordable treatment for people. What an awesome interview thanks again so much. Megan for taking the time to chat crisper genome editing with us. We loved it and Aaron we should definitely do an entire episode on crisper someday. All right should we do sources? Yes absolutely. Okay. So four my sources I read a few books. One is called soul the Black Panther party in the fight against medical discrimination by. Nelson. Also, as I mentioned earlier, killing the black body by Dorothy, Roberts and dying in a city of the blues by Keith Way Lou. and also I have a few other books and papers that I will link to couple of papers I WANNA shout out our by steen's Ma at all Walter Clement knoll first patient described with sickle cell disease and by Barasch in nine hundred ninety eight sickle cell trait policy and research. Paradigm 's awesome. I. Read a few good book chapters that I will link to as well as there is a great sickle cell disease in Nature Review Disease Primers. That was from twenty eighteen. If you want just kind of a nice overview of the biology of sickle cell disease, and then if you want that paper on the comparison of funding between sickle cell, an cystic fibrosis was by Fahim for rock at all published in Jama. In twenty twenty just earlier this year we post all of these sources as well as the sources from every one of our episodes on our website this podcast will kill you dot com just click on the episodes tab will thank you again so much to the amazing Marcia and Cherie for sharing your experiences with us and to Meghan for walking us through the incredibly cool world of crisper technology. Yeah. Thank you all so much and thank you to blend mobile for providing the music for this episode and all of our episodes and thank you to you listeners for listening. We love you. We appreciate you. We hope you like this episode. Yeah it was really fun. So we hope you had fun too. Until, next time wash your hands you filthy animals..

sickle cell trait Nature Review Disease Primers social solutions America NIH Megan Walter Clement knoll Aaron Keith Way Lou. Gates Foundation Nelson Jama Fahim Barasch Meghan Ma steen Marcia
"sickle cell disease" Discussed on This Podcast Will Kill You

This Podcast Will Kill You

10:09 min | 9 months ago

"sickle cell disease" Discussed on This Podcast Will Kill You

"And these acts. These discussions of course resulted in accusations of restricting black fertility, racial genocide, and new eugenics and rightfully. So yeah. Those with sounds like to me Oh. Yeah and this this misleading reproductive counseling for sickle cell was just one way that reproductive restrictions were intentionally or forcefully placed upon black people. I really recommend killing the black body by Dorthy Roberts to read more about that topic and. So before wrapping up with the history of sickle cell in the nineteen eighty s and nineteen nineties, I, WanNa read a quote by the author of dying in the city of the blues that I think does a really good job of summing up the nineteen seventies and sickle cell. Perfectly. The story of sickle cell disease in the early nineteen seventies also revealed the ways in which the political process both channeled and deflected the popular activism of the time. It was a time of grudging recognition of the black experience, but it proved difficult to translate that awareness directly into health policy without creating enormous new stigmatizing burdens for black Americans and without fostering growing cynicism about racial politics. Yep Yep. And so that brings us to the nineteen eighties and nineteen ninety S I don't WanNa. Step on your toes too much Erin about whatever you WanNa talk about. So I'm just GONNA go over a few big developments or patterns that emerge during this time with regard to sickle cell. that. I. Have a feeling. You'll talk more about. Okay. Let's see. Yeah. So as you as you mentioned, pain management is a huge component of sickle cell anemia and the sympathy for people with with sickle cell that seemed characteristic of the nineteen sixties and nineteen seventies. Kind of gave way to this disturbing trend of cynicism and stigma. More and more healthcare providers seem to simply not believe that people with sickle cell anemia were experiencing a true painful episode and there were increasing reports of healthcare providers accusing their sickle cell anemia patients of faking it of exhibiting drug seeking behavior, and correspondingly limiting the pain medication prescribed and. Earlier when you talked about the different time line of when at different ages you experience, you're more likely to experience one symptom over another that the the increase in painful episodes in late adolescence and early adulthood is something that also like. Made this whole made this whole thing worse they were like, oh well, you're young adult you're just seeking drugs so I'm not going to give you any oh my God, and this is you know this is despite the fact that there was research indicating that this wasn't going on that people with sickle cell anemia were just as worried about their own you know narcotic consumption or pain medication consumption as as anyone else in it was it's just like it didn't seem to make a difference. Yeah. It it. Seemed to be like this, this belief that became so embraced and like so difficult to get rid of it. So frustrating that in so many papers that you read it's still something that is mentioned like Oh you often have to use opioids to treat pain which can lead to addiction. It's like that's true in anyone and there's no higher rates of opioid addiction in people with sickle cell anemia in the general population like they're just isn't. So it's it's infuriating that you'd be withholding treatment that is necessary. ooh. Yeah well, and there's also there was also something that was mentioned in this book about how there is research indicating that opioid addiction starting from hospital treatments or medical treatments is extraordinarily low that is not the way that the vast majority of deductions begin and. But despite all this, this enormous bias still remains and this is I mean, this is a a larger issue. Yeah the. Ability of pain medicine, we can't measure it and I think that makes that makes people trust it less trust to the person lesson is sort of goes back to what I was saying earlier about how like medicine became more about the body measurements and these things that you could you know. You could put on a chart then it became about the person's experience itself. Yeah. So yeah. Yeah. But in this context, what this meant this, this increasing, you know disbelief was that people with sickle cell anemia were at renewed risk for their pain their experienced to once again be neglected, ignored and made invisible. And this persists today this issue. Yeah and these decades also brought the promise of many different therapies for sickle cell anemia such as hydroxyurea as you mentioned and bone marrow transplantation which didn't necessarily up. The shiny promises that had been made about them in their first introduction. But I'm really hopeful to hear more about. New, approaches. But I want to end now again with other quote again from Keith Way Lou the author of dying in the city of the blues. FOR LIBERALS MODERATES AND CONSERVATIVES ALIKE The history of neglect and the diseases chronic painful character seemed to reflect white America's neglect and misunderstanding of black health concerns and demanded attention. The disease became a multipurpose metaphor, a proxy in social, economic and political debates about a wide range of seemingly unrelated issues. Okay Aaron bring me up to speed on what's going on with sickle cell today. Okay. Let's take a quick break I. Are Eight. So. We'll talk I doubt numbers, how many people are being affected by sickle cell. In the US and in the world today, and then we'll touch a little bit more on why that is and the malaria connection because I do think that's a really interesting part of the story. And then we'll talk about current research. Does that sound good sounds great. Okay. So we'll talk first about the US and then globally. So in the US newborn screening is conducted since two, thousand, six or two, thousand, seven across the board in all states plus Puerto Rico in the US Virgin Islands So we know the rates of sickle cell a Lille in the population. So one copy having one copy of sickle cell trait. So. Overall in the US in two thousand ten, the incidents was fifteen per one thousand babies born treat trait. Okay. But this is a huge range from seventy three per one thousand among black newborns to. Two. Per One thousand in Asian native? Hawaiian and Pacific islander newborns three in white babies and seven per one thousand in Hispanic newborns. And this is voluntary or mandatory screening. Soon, newborn screening is generally it's Universal I. Think it is possible to opt out of it. But in general, it's universal and kind of recommended I think most of the time. They don't want to leave the hospital without newborn screening. Because it doesn't only screen for sickle cell the screens for a whole bunch of different. We talked about this in a cystic fibrosis episode as well. Right right. Because you're also identifying then those newborns with sickle cell anemia. But what's interesting is that it's actually hard to get a number on the number of his in the US born with sickle cell anemia, which I think is interesting. So let's talk about the whole globe. How many people are born every year with sickle cell anemia. Globally estimates are about three, hundred, thousand just over three hundred, thousand babies born every year with sickle cell anemia. That's a huge number. It's a massive number and I want to point out that that number is the number of babies born with sickle cell. H. B. S. S. but remember that there are other ways that you can have sickle cell anemia rate. You could have it with one copy of H. B. S. and one copy of Beta thalassemia. You could have it with one copy of H.. B. S. one copy of HP see those. Aren't included in that estimate of three hundred thousand. So okay it's thought that that total number accounts for about seventy percent of the total amount of sickle cell disease. So that whole range of clinical disease. Worldwide and it's also estimated that about half of these babies worldwide are born in. Nigeria. The Democratic Republic of Congo in India. And that in many parts of sub Saharan Africa. Sickle cell anemia might be responsible for as much as six percent of all childhood mortality. Six. percent. Oh. My God just from sickle cell anemia because in many places in many parts of the world. Under five mortality from sickle cell anemia. So dying before your fifth birthday can be as high as fifty to ninety percent, which is atrocious. Wow..

sickle cell trait US pain Dorthy Roberts WanNa H. B. S. S. Saharan Africa Democratic Republic of Congo Erin hydroxyurea America Nigeria malaria Aaron HP thalassemia Puerto Rico India
"sickle cell disease" Discussed on This Podcast Will Kill You

This Podcast Will Kill You

09:38 min | 9 months ago

"sickle cell disease" Discussed on This Podcast Will Kill You

"To enter. My Name's Marsha. The UK can tell from accent. And I'm I had sickle cell for the longest time I can remember. My parents found out when I was a foul. Six years. Old. My mother knew she had the trait. But. My Dad didn't know that he carried the try to see my oldest of this bone. and. She just had a trade said it was among was fun. She left me. When I came about I was known with which is called V6 deficiency. So it kind of must. The sickle cell. So. Many vs are not always becoming sick that couldn't workout. And then. When my youngest was test for the south and that's when they found out that. I had. The full blown disease. kind of on the stood. I had 'em much later on I would say when I was about. Eight or nine. Yes. I went to hospital put Minsk prior to that but it didn't really sunk in a kind of let my parents deal with it all like a cable you manage my health on being a child than playing with my friends and stuff like that I didn't really understand it too. I. Think Lights Martine years it started gun secondary school. That's when is that taking more control the my illness sign will. have to eat I have to dress Ri- a have to make sure I get enough rest and. Restrict myself because on these things can trigger off a crisis in I condemn. weeks on end and those are me when I'm joined. So black. Be. Positive Choir but I actually came out and I love my school friends. Didn't I did not know you had six without. Did Not have to explain it to them in in a way and I did know how they say is always think like you know try set. Loose friends which are member having the frankly said, they didn't want to be my friend because they feel like Pepsico so if they held my hand. And a neat doesn't let that way it does not work that way I. It was very. Ah Shit a shield myself. So other than. That next ticket city in that. Light to on my adult. Years. You know if you don't like me the way I am than. Fine somebody else world as many people in this world that will and I grew confidence and Zyppah Tae. teenage girls and boys who were in my situation I. Don't Let. Yourselves stop you from doing what you? WanNa do. When I do get in a cost in paying. The best way to describe it has to be like. When you get a really bad cold and you have X. Men's. And Olivia Padilla. On a ten times was scaled aches and pains. Nico called. And you just want it to stop and I had exposed me to tears before many times and will be part swear did not don't WanNa. Be here on this earth because I don't want experience pay why do I have to go for? You know why was the unlucky one? And I did come go to the sites of blaming my parents a you should check teacher before you had me watching you do this. On your mind. Starts Thinking loads of things of united apply. If I wasn't hey, would I be in the place or if I was bullied before almost to stuff redoubt being a better place, you just think many things. Every day. You had cup. You don't know if it's GONNA. Be a day if it can be bad day. I. Think that affects your social life is while. y'All counseling on your friends and it's same with relationships. Like br account with a low of companies because they don't understand the extent of sickle cell got into hospital August result on our mind retentive. Know why got into hospital but we like Google trait the best we can hire sometimes we don't like on hospitals because of the stigma that we get, we get on as a drug addicts. Will. Really pay what just because we need a fake. Don't want to be in. IS THE LAWS PACE GONNA be. Lucky enough. My family are amazing. Bless their hearts and a half snapped them many times and Is Not Hesse remain to is just a case of. I can conceal is this pain and I don't WanNa feel the pain anymore and I have apologised them off Louis but now conscious no by rotating. Vanessa minute crosses that gun questions they just. Payments. Heat pad the all hope. Is that bad you need to go to the hospital and of the state of the Sun. is fantastic. He a code Quinn the minute you know Mommy's not feeling very well, he's on it with the mets, the cups of tea the wounds also everything he can to mate to cheer me up you put my favorite movie on and cockles. we've made said he initially amazing but because it's just made him that lifts giver I feel like that. Sometimes he feels he's childhood robbed in some titans. He does he can't be a child because he has said end will say big himself, which is on new is quite hard nothing. That was the same thing when I knew when I was going to have children, you know what impact? Would my health have on him and? Me when I fell pregnant on often my pregnancy that's when my sister. So what was I had to monitor stroke most insulin happing to my body. Felt was tailgating. Field that. That was not made. To me when I was thinking about slot in family. So I'm now governor experiencing things where a fill that may be. I could be night mode aware of other given the option. Of what today but nevertheless? A Latin spaces? Think Lau that way I. Joined be positive choir. Is Journey that you don't want to end singing for. Britain's talent singing. The crane. Mega William one it was amazing. It was living. The dream had to keep saying Pinch Mason. is at mechanism that. It was actually amazing the way since we've come on that platform is gone more awareness everybody sutton to get involved starting to. Be More clued up in taking notice of will secrets out. How they can go around helping to spread the awareness, an NFL that. Away that brings the community together as well. 'cause in the quiet there are many people who have the illness and way shea also raise week in everybody experiences differently that is. Where we've become like a big unit. And get to share it around the world which is amazing and we have enough we have a lot and that's the main thing. Yes. You have your damn days but also you have your good days. But everyone. Is it. You're always smiling on the think allocate the positive that. sickle cell as a as update full. Actually see as a gift and a blessing to have because I can go out with the about six. And make weddings. Yeah. Unhappy initially afraid I couldn't be in will happen. So enemy Sherry to Soviet if. I was born in Uganda is more east African country. And UH. Sickle cell disease. So when I was my mom and dad had it varies of story the buster that baby boy like appearance or very excited. Haven't baby boy I.

WanNa Pepsico UK Minsk Nico Sherry Ri Olivia Padilla Uganda Vanessa Google titans tailgating mets Hesse Lau Louis Quinn Pinch Mason.
NFLPA tells agents to inform players of virus risks

Golic & Wingo

03:05 min | 11 months ago

NFLPA tells agents to inform players of virus risks

"The NFL Players Association has instructed their player agents to talkto all of their clients about the Ricks risk factors that could make them more susceptible to severe illness as the result of the Corona virus and Among the things they talk about Mike in this letter players who may suffer from chronic kidney disease or COPD, chronic obstructive pulmonary disease, right immune compromised state weakened immune system from solid organ transplant. Serious heart condition. Sickle cell disease type two diabetes. And then there's this one Mike, which is the first place I went when I saw this list. Ah, B m I r a body mass index. Of 30 or higher and let me be clear, according to the BM. If you have a body mass index of 30 hire your clinic are technically obese. Now. A lot of people would have some disputes with that, and I can understand why. But if you're talking about a B m E of 30 you have just described literally. Every single offensive lineman currently signed to a roster probably even go lesson that toe linebacker and or tight and I'll get to the heights and weights in a minute and understand when they had say all these things. It's not only the players, but remember, the NFL is not going to be in a bubble, so players are going to be going to practice. Flying two trips and then coming home to family. So again, you're going to be involved in, you know, are you single that may have have Ah say on if you're going to play You're married. You have young kids, You know that You're coming home to every day from practice or from runaway trip. So And what if some one of those kids you know if they have a weakened immune system, Do you want to take that chance? So this is not just the player himself, but also the family as well. But as Faras, the bm is now there are tons of charts author. I just kind of picked a chart that basically be, am I, you know Everybody understand. You know the height and your body weight your body Mass. So it gives you how how tall you are and the wait for your B and I, And there's a lot of discrepancy about there is soul. Listen, As I said, I picked up a chart. I'm sure people will tweeted ecological lingo, different charts. If you can't great because I'd like to see some different ones because some of them you look at our like, Wait a minute like the one I'm looking at right now. Says last I checked a six foot tall is 72 inches, Correct? Yes. Yeah, Okay. Just wanted to make sure that your question is good If you're six foot tall and you weigh £221 You are right at 30 bmo. You're right at the obese, which is ridiculous. If you're and then just keep going up because you start look at tight ends. Linebackers. Obviously, offensive lineman. They're usually that you know, anywhere from six to toe got 68 Let's go to like 65 Okay, six foot five. Or 64 76 inches, 64 If you weigh £246. That's beyond my 30. According to this chart, you are considered a

Mike Nfl Players Association Copd Ricks NFL Faras
Doctors Have Injected DNA-Editing CRISPR Into a Live Person's Eye

Short Wave

01:13 min | 1 year ago

Doctors Have Injected DNA-Editing CRISPR Into a Live Person's Eye

"Tell me why Chris so awesome. It's really cool because crisper. Is this really powerful? Gene editing technique that allows doctors to make really precise changes in our genetic code. And so it's got incredible potential for treating lots of diseases right. It is actually not an exaggeration to say that crisper could revolutionize medicine in future in today. You have brought us another crisper milestone yet. Mattie get this for the first time scientists have used crisper to try to editor Jean while the DNA is still inside the body which is wild because until now offer crisper treatments for things like cancer or sickle cell disease. Doctors had to remove the cells. They wanted to change. The patient's bodies edit them in the lab and then put them back in right for this time. Hopefully the editing will take place inside the I of a patient who is almost completely blind due to a genetic disease. Called Lieber Congenital Amoroso's the experimental treatment was done by doctors at the Casey Institute in Portland. Oregon. The hope is that the crisper will edit or fix the mutated gene that causes the disease and potentially restore the patients

Mattie Casey Institute Chris Lieber Amoroso Portland Oregon Cancer Jean Editor
"sickle cell disease" Discussed on Faith Health & Home

Faith Health & Home

10:55 min | 1 year ago

"sickle cell disease" Discussed on Faith Health & Home

"Hello and welcome to the faith health and home. Podcast joining me. Today is Dr Ted w love. President and CEO of Global Blood Therapeutics to disgust disproportionate effects of sickle cell disease in the black community and the new FDA approved options available to help manage the disease. He also how African American doctors have been instrumental in changing the landscape of sickle cell to give patients and their loved ones a new fouled. Hope stay tuned faith. Health and home is coming up next. Welcome to the faith held in home digital podcast. I'm your host Makeba Giles. Here we share information and resources for physical emotional and spiritual being to help families live and inspire lifestyle and encourage healthy living. Thank you for joining us. Black history month is when we focus on trail blazers in the African American community. Ev includes medical pioneers. Who have made an impact on conditions like sickle cell disease a genetic blood disorder that this proportionately affects the black community although the disease was discovered one hundred years ago. It has historically lacked treatment options. That is until now joining me. From San Francisco to discuss the latest advancements in managing sickle cell disease is Dr Tale Love President and CEO of Global Blood Therapeutics a biotech company based in San Francisco. Thank you for being with me today. Dr Thank you for having now on first doctor you fall. On the footsteps of other trailblazers in the world of sickle cell disease what are some of their contributions to our understanding of sickle cell? A great question and we have made progress in sickle cell disease into positions african-americans. Vision that I'd love to mention our doctor. Charles whitten from Michigan and Maryland Hughes Capstan. Both of those physicians recognized in sickle. Cell disease was primarily in their day killing individuals killing patients at very young age and they recognize that it was actually infections. Typically PNEUMOCOCCAL infections Dragging the early debt. And it was through their work that we recognize that if from screening for sickle cell disease and we offer simple things like antibiotics and pneumococcal vaccination. We could prevent bills premature deaths and they were writing now. Sickle cell patients rarely die as children. they live typically in the forties So that's why we now need the next breakthrough of innovation. We've been able to get from survival in the teams to survival in the forties. But now we need to go after what is driving the cause of death in the forty. What's driving the disease so that we can powerfully? Go after them and that's really where we are and that's really what our company is focused. Absolutely now you have worked as a physician for many years. And now you're on the Bio Pharma side of the industry Can you tell us? Is that why you came out of retirement to continue your work? I can't and as you said I've had a long career as a physician science and as a result of that I was able to retire relatively young and I moved to the wine region North of San Francisco But I got a call. One day From the founders of global blood therapeutic and they said we have an idea that could fundamentally go after sickle cell disease at its root cause and change outcomes powerfully so I looked at the finance and scientists one of the things that I do understand pretty well and because I retired. We had a family meeting. And when we discussed this other family are two daughters literally fed that you have no choice. This is something that could be very powerful cell patient and this is something that could be powerful for our community our African American community so I came out of retirement because this was very personal to me. It's very personal to our family and it has been the most wonderful paying that I could have done in my life really focus on helping patients that need help helping patients that have been ignored in providing a very powerful solution to a very serious bomb in. That's wonderful that you Took that passion to decide to come out of retirement to help others as greatly appreciative for all that you do. Now there several misconceptions associated with sickle cell disease Can you explain some of those and why it's important particularly for the African American community to be properly informed? I sure can. So you know the sad thing is that many of the misconceptions of sickle cell to these are actually Jew by the devastate aiding nature of the disease so one Stereotyped sickle cell. Patients are lazy They'RE NOT LAZY. They just don't have blood. If I took half of our blood out we would not feel. We would not have the energy or the stamina to get through a full day of normal activities so these patients are profoundly anemic because of it the and sadly they've been stereotyped as being lazy because on the consequence of their the the other big stereotype. Is that big are drug. Seekers and in fact sickle cell disease is causing the red cell body To be destroyed and the red SOx content liberator our blood softens that causes swelling blood vessels which results in cells sticking together and closing off blood vessels entirely. That's very painful. But you imagine attorney on your finger. How POWERFUL HOW POWERFUL PEOPLE. That would be so. These patients have been labelled as drug seekers when in fact they are seeking relief of some of the most excruciating pain that you can ever experience but despite that they'd been labelled as drug seekers when they're just seeking relief from catastrophic catastrophic consequence of sickle totally and absolutely now and we mentioned earlier their progress has been made in the research and management of sickle cell disease over the years. I'm tell us what kind of options are now. Available for people. Living with the disease last year was have made in your sickle sell in the United States. The FDA actually approved not one but two novel drugs that are specifically aimed at treating. The problem sickle cell disease. The first one but I'll mention was developed by a company named Nevada's That drugs is an intravenous drug that Taken once a month and it's designed to block the inflammation in the blood about that. I mentioned which are leading to the exclusion of blood vessels as measured by these Basil occlusive. Crises are paying crazy so that was approved in early November of last year. Literally a few weeks later the FDA turns unapproved drug and drug is unique in that it was approved to treat the fundamental problem of sickle cell. Disease that in the hemoglobin organizing these rods which causes the fell sickle which causes them up. Sure which leads to all the downstream consequences and because of what our drug does he was. Actually the third strug received breakthrough designation for sickle cell. Disease never happened before south and it was also the first drug to receive accelerated approval for sickle cell disease so big innovations big recognition by the FDA that if you can go after the fundamental basis of the leaf. That's how you can make changes. My malady would be HIV. We used to treat the infections called H. Though when the big breakthrough came from we actually treated the various. So we're now developing therapies. Which are gone yet. The root causes sickle elderly as opposed to just focusing on the downstream consequences. That's where you're gonNA make big innovation and that's where you start to make people that have normal. Life expectancy and to be very well managed indeed the nets phenomenon that those treatment options are now available. Four people in great to get that information out there so that people can know In addition to those treatment options that you just mentioned I'm well some things that people who are living with the disease can do on the day to day basis With their daily lifestyle to help manage sickle cell. Well I think there are I. I always thought that knowledge is power and I think the number one thing particularly with the Internet now is that a lot of resources that sickle cell patients and families should be going to learn about not only our innovations in therapies but about how to manage their disease overall. So a couple of flights that I would mention Sickle CELL SPEAKS DOT COM. That's www that sickle cell speaks dot COM Meta information about our novel medication Ox Brighter can be found on option provided DOT COM. That's O. F. B. R. Y. T. Hey dot com. And then we have the sickle Cell Disease Association of America Rate Website which is sickle cell disease dot. Org thank you for sharing those now. I talked to singer and actress. Jordin sparks awhile bag. She shared her personal story of how devastating.

Disease sickle Cell Disease Associatio Cell disease San Francisco African American community FDA CEO of Global Blood Therapeuti blood disorder Global Blood Therapeutics PNEUMOCOCCAL Makeba Giles Dr Ted w Dr President and CEO President Maryland Hughes Capstan Bio Pharma Nevada
Leading St. Jude's Genome Engineering Capabilities: A Chat with Shondra Miller

CRISPR Cuts

08:38 min | 1 year ago

Leading St. Jude's Genome Engineering Capabilities: A Chat with Shondra Miller

"Welcome Shandra Thick. You must be here all right so I would like to ask you about what you do. Junior current research on. Maybe about a little bit about your background as well. Yeah so I've been doing engineering. Basically my whole scientific career so I did my PhD on Singer. Nicholas which is kind of like the grandfather of Christopher Gas Ninety Will Matthew Portas lab and so we were at the time just doing protein engineering trying to get zinc finger. Proteins NUCLEAR ACES ACTUALLY. Cut some specific site in the genome. And it's sad thing now but I spent five years basically making a pair of glasses that gop so very exciting at the time but it really became outdated and so then after graduate school. I went directly into industry. I worked for Sigma Aldrich on their composers of technology. Where we're doing the offense and it was exciting time in genome editing. Because for me especially because I had been doing protein engineering and then I was able to go to sigma and they would give us the proteins and we actually do genome editing for the first time really. I was able to like okay. I want to make this point mutation. How do we do it? And we were able to do. Some of the first different types of editing so like with food chain Chen we published a paper. Where using singles trying to all go donors. Almost everybody uses now. We were one of the first to do that. We can do with high efficiency losing figuring places and so then in two thousand twelve so pre crisper still. I decided that I wanted to start. Asia resource at the Times inferior increases. Were so expensive. That people weren't really able to access them especially in academic so I think the Times in cases where like twenty five thousand dollars just to plasmids so you couldn't actually do your editing editing to get yoursel- liner animal model. So it was frustrating to me is like this is game changing technology. People people need access to this. This is going to change science as we know it and so I actually went to Washington University and I put the idea of starting to share resources. Said you know. People don't want to become genome engineering experts. They want their cell line. They WanNa make do a screen. They want their animal model. They WANNA continue asking the questions that they are experts and they just want the tool for the most part. So let's start a resource. Let's be the genome engineering. Experts will help other people expedite science at Wash U. in and around the world and so we started that it's called the engineering IPSEC center. It's still going strong G. I see there and then in two thousand seventeen. I got a call from Saint Jude. And they were like. Hey we want to start something like what you're doing that Wash U. as I said no I'm not interested at the time I was actually six months pregnant with my second child. I'm had started This core the see from there was like my first child. Not My biology child. Something that I cared about the people and what we were doing so it was really not very interested in. They said we'll consult for us in How to get this thing going and short story long. I guess I ended up moving to Saint. You'd because it's kind of a really special place to do research as like a magic fairy tale land to do research especially for a shared resource so we actually given dedicated. Rnd Time and we're institutionally subsidized and so on most Groups Shared resources pay their own way so basically the services that they provide pay their salaries paid for the reagents that they're using but at Saint Jude. They say no. We're GONNA guarantee your salaries we're gonNA give you money to actually do the science that you need to do to help everybody at Saint Jude. So you don't have to worry about that. Just do good science. So that's very freeing. And then the ability to have a dedicated are indeed budget. So we're like okay. We're going to not only do genome editing. For other people were GONNA add back to the field and and keep at the forefront of it so I know that was a really long answer five. That's how backgrounds and doing a lot of genome editing. The events Talen crisper over the last fifteen years or so. That's great. Could you tell us about a couple of your research projects that you have gone? Yeah so we do a lot of things to improve efficiency. We have some ideas about how to get longer inserts and two so one of the things that's really hard crisper is when you're trying to knock something in fairly easy to do something. Small appoint mutation or five tag but it gets more difficult as the insert increases so Rachel Levin in the lab. She's working on how to get longer inserts and increasing their overall rates of HDR something. Saint Peter's in the lab is working on some strategies for enriching the donor concentration in the nucleus. And I think one of the most exciting things that we're working on is a cure sickle cell initiative basically. Where they're you know. Saint Jude. It's a research hospital. We actually have our large sickle cell population in Memphis. I think there's about eight hundred sickle cell patients that we see at Saint Jude. And so send you actually partnered with several other groups not only at Saint Jude but also outside of Saint Jude. So we basically worked towards making therapeutic for sickle cell disease and Mitch Weiss Option Darsi and myself from Saint Jude Consortium. And so we're we're really looking forward. Hopefully going to a therapeutic In the coming years and so that's why the more exciting things that we're doing this more closely related to the Translational medicine wonder. How can you tell us a little bit about just different technologies age with Chris? Frightening so you tried like classmates. How did you come across it? The goers that Rene has ever feel y'all so we actually remain your own guide. Ernie's for five years. We WOULD MOSTLY DO PLASMA. Cloning where we played together you cut the backbone with type. Two S restrictions. I'm basically pop them in like into the planet and that works but it takes probably like four hours of hands on time ish or of about three to five days and so whenever we saw that you could do synthetic guides I guess the limitation was people were able to synthesize the long Arnaiz right. So I think that that's where something kind of burst on the scene and so that we saw that was attractive to us but we needed to be able to do it quickly right so we can make these platforms fairly quickly. Do also do. Ib Tea and make that fairly quickly. The synthetics at the time you know there are companies that were doing it but it was very expensive. You get quite a lot and we don't really need a lot. We have a lot of smaller projects that we're doing. It took too long for us right. So we're trying to make cell lines for folks. We're trying to make animal models for folks so we don't have to wait a month to get the guide so that's why we were making them ourselves so so they go came on the scene. I think we started. We made our first order to go in October. Two Thousand Seventeen. Basically gave us a quick turnaround time. I think we were like Beta testing small to smaller scale early on and so from then. I guess this history of doing this. Cloning bacterial colonies and doing many. Perhaps you're able to do make more cell lines and made more animal models and do more research. Christopher is a relatively young technology but it's made a pretty big impact as you've mentioned already. What do you think the next five years will look like in? Where do you think the field is heading? That's it's an interesting question. I guess there's two or three or four different areas that you think about I think about basic science and experimental biology. How can we do things more efficiently to make cell lines that people can understand biological processes or ask for biological question? So I'm usually thinking about research purposes. I think it's only getting faster. More efficient and cheaper which is really democratizing research. A lot of different ways I think crisper has been probably the biggest discovery understanding how to do crisper since like I think it will science face a science as much. Pr Has Changed Science. Maybe more you think about therapeutics there'd be trial we're going on with Christopher Pasadena. Think people starting with blood stuff because they can get to the stem cells in their starting with is stuff because they can get to the right cells so I think one of the biggest limitation to Crisper as far as there is delivery. How do you get it to the right cells So I think that's a limitation? I don't know I guess I would really say the sky's the limit just the ability to be able to go into complex genome and make precise modifications huge. I think we're only going to get better S. nine in all the variants or the logs. Pretty darn good. We worry about off targets for research purposes. Were not outright about it. Because you know. We're studying a question for their buicks really important. But we've made such big advances since you think about two thousand twelve two thousand thirteen. It's just it's a whirlwind and you think about how you know. I think the offense really paved the way. But you know the offense where we use a million tells like nineteen ninety nine two thousand three but took two thousand three two thousand ten before we even Pat Talents and then new talents has a very short heyday. And then Chris Brazil Blown on the scene. I think there's huge diagnostics agriculture therapeutics. It's just it's kind of unlimited possibilities so I think we'll see in the next few years and we're seeing now. A lot of ethical questions people are asking. What should we do because we can do a lot of things? But what should we do? I think that that's one of the things that people will be discussing over the next few years

Saint Jude Chris Brazil Sigma Aldrich Shandra Thick Saint Jude Consortium GOP Nicholas Washington University Memphis Asia Mitch Weiss Christopher Gas Pat Talents Translational Medicine Rachel Levin Matthew Portas Chen Ib Tea
CRISPR For Sickle Cell Disease Shows Promise In Early Test

Michael Medved

00:26 sec | 1 year ago

CRISPR For Sickle Cell Disease Shows Promise In Early Test

"Sickle cell disease is an inherited blood disorder affecting about a hundred thousand Americans mostly African American it's a debilitating condition causing in the media delayed growth vision problems and painful swelling and hands and feet because the organ damage from sickle cell disease shortens the lives of patients there is ongoing testing to see if a deck video might address that too I

Blood Disorder
"sickle cell disease" Discussed on AP News

AP News

11:17 min | 1 year ago

"sickle cell disease" Discussed on AP News

"There's a new drug on the market to help patients reduce the number of pain episodes they suffer with sickle cell disease flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

09:13 min | 1 year ago

"sickle cell disease" Discussed on AP News

"Flare ups of sickle cell disease are extremely painful and damaging when red blood cell stick together blocking blood from reaching organs and small blood vessels the new drug from Novartis is called a dock Veoh during testing it reduced pain episodes in half for about 200 study participants researchers are still testing whether it might help extend the life of sickle cell disease patients possible side effects include influenza and high fever and the drug costs more than $80000 a year I'm Jackie Quinn

Novartis Veoh influenza Jackie Quinn $80000
"sickle cell disease" Discussed on AP News

AP News

14:31 min | 1 year ago

"sickle cell disease" Discussed on AP News

"US health regulators have approved a new drug to help reduce the pain of sickle cell disease the food and drug administration's approved a doc fee for patients 16 and older it's given as a monthly infusion and cuts in half the occurrences of sickle cell pain episodes but the drug from Novartis AG is pricey between 85000 and $113000 a year sickle cell disease is an inherited blood disorder affecting about 100000 Americans mostly African American it's a debilitating condition causing in the media delayed growth vision problems and painful swelling and hands and feet because the organ damage from sickle cell disease shortens the lives of patients there is ongoing testing to see if a deck video might address that too I Jackie Quinn

Novartis AG blood disorder Americans Jackie Quinn US $113000
"sickle cell disease" Discussed on AP News

AP News

14:31 min | 1 year ago

"sickle cell disease" Discussed on AP News

"US health regulators have approved a new drug to help reduce the pain of sickle cell disease the food and drug administration's approved a doc fee for patients 16 and older it's given as a monthly infusion and cuts in half the occurrences of sickle cell pain episodes but the drug from Novartis AG is pricey between 85000 and $113000 a year sickle cell disease is an inherited blood disorder affecting about 100000 Americans mostly African American it's a debilitating condition causing in the media delayed growth vision problems and painful swelling and hands and feet because the organ damage from sickle cell disease shortens the lives of patients there is ongoing testing to see if a deck video might address that too I Jackie Quinn

Novartis AG blood disorder Americans Jackie Quinn US $113000
"sickle cell disease" Discussed on AP News

AP News

14:31 min | 1 year ago

"sickle cell disease" Discussed on AP News

"US health regulators have approved a new drug to help reduce the pain of sickle cell disease the food and drug administration's approved a doc fee for patients 16 and older it's given as a monthly infusion and cuts in half the occurrences of sickle cell pain episodes but the drug from Novartis AG is pricey between 85000 and $113000 a year sickle cell disease is an inherited blood disorder affecting about 100000 Americans mostly African American it's a debilitating condition causing in the media delayed growth vision problems and painful swelling and hands and feet because the organ damage from sickle cell disease shortens the lives of patients there is ongoing testing to see if a deck video might address that too I Jackie Quinn

Novartis AG blood disorder Americans Jackie Quinn US $113000
"sickle cell disease" Discussed on AP News

AP News

14:31 min | 1 year ago

"sickle cell disease" Discussed on AP News

"US health regulators have approved a new drug to help reduce the pain of sickle cell disease the food and drug administration's approved a doc fee for patients 16 and older it's given as a monthly infusion and cuts in half the occurrences of sickle cell pain episodes but the drug from Novartis AG is pricey between 85000 and $113000 a year sickle cell disease is an inherited blood disorder affecting about 100000 Americans mostly African American it's a debilitating condition causing in the media delayed growth vision problems and painful swelling and hands and feet because the organ damage from sickle cell disease shortens the lives of patients there is ongoing testing to see if a deck video might address that too I Jackie Quinn

Novartis AG blood disorder Americans Jackie Quinn US $113000
Seattle Children's Hospital opens Building Hope expansion

Morning News with Manda Factor and Gregg Hersholt

00:38 sec | 1 year ago

Seattle Children's Hospital opens Building Hope expansion

"New facility is opening at Seattle children's hospital to offer more life changing therapies for children who are battling disease it's called building sure and this is Seattle children's CEO Jeff sparing with this new building alone now will have more than a million square feet of research space dedicated to kids again one of the five largest in the country and we're really proud not only what we're doing now more importantly the fears that are going to come in the future because of what happens in this whole children's is one of the nation's top five pediatric research centers and is dedicated to developing better therapies for diseases like cancer sickle cell disease A. D. H. D. and

Jeff Seattle CEO
In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

All Things Considered

04:14 min | 1 year ago

In A 1st, Doctors In U.S. Use CRISPR Tool To Treat Patient With Genetic Disorder

"For the first time scientists have used the gene editing technique called Christopher to try to treat a genetic disorder in the United States NPR is the only news organization to have learned the identity of the first patient and to talk with her NPR health correspondent rob Stein is here with more he rob ARE who is this woman and why she undergoing this experimental treatment her name is Victoria gray and she's thirty four lives in force Mississippi with her husband and four kids and here's a little bit about what you told me about herself when I met with the recently at the Sara cannon research institute in Nashville well I don't work out stay at home mom but before I got to see I was working in the beauty department at Walgreens and I was going to school to become a nurse but all that that put on hold for health reasons tell us about her sickness what is her condition yes of Victoria has sickle cell disease and it's a terrible genetic disease that primarily affects African Americans in the United States instead of having a normal red blood cells you know the cells to carry oxygen in your body sickle cell patients have a hard sticky the sickle shaped red blood cell that causes Carol bouts of agonizing pain and cost lots of really serious health problems well in high risk for stroke thing on Harry's full hearted takes in these things can happen to me in a blink of lack in not paying epistles can just come on after the blue I can just be laughing the next meeting of crying you know in some of the worst pain that you could ever imagine is a heavy load to carry he now that sounds really tough so how are doctors trying to use crisper to help people like Victoria gray right so this is how it works to doctors a cake sells out of the bone marrow sickle cell patients and they use this crisper editing tool to edit a gene in those cells to turn on the production of something called fetal human which is usually only produced by fetuses when they're in the woman babies for short time after they're born and then the infused billions of these genetically modified cells from the patient back into the bodies hopefully to help treat the disease doctor harder Frankel was running the study natural he explained a bit more about how this is going to work what we are trying to do here is we're trying to into use enough fetal hemoglobin into the red blood cell to make the red blood cell go back to being happy squishy and not sticky at heart and can go deliver oxygen what it's supposed to so she is the first sickle cell patients to get this treatment what does it involve what was the process like yes parts were really pretty heart shape to go through chemo first of all white at our own bone marrow to make room for these Christopher headed cells and then doctor Frankel infuse more than two billion of those Jeanette itself into our body this is just a few weeks ago they had the sales in a big experience and when you went in here my heart rate shot up real high and so there was a little scary tough moment for me news at that Iraq rival deals had PC use was that feeling like it was amazing and you know this kind of overwhelming after after on then I hate went through to find me you know D. what I came for her and for tour she calls her new Jeanette itself because of her supercells Whitey gone supercells well I have sickle cell so this place would better AS is this prevail Robert sounds like there's a lot of potential here but one of the concerns yes you know they're always concerned about any new experimental treatment is it safe will it work and this is all really magnify was something that's this new hears of Laurie Zoloft she's a bioethicist at the university of Chicago not talk to her about that I am optimistic about the success of Christopher I just want to be done carefully yes so they're gonna monitor very closely first of all make sure the edited cells are safe not causing any health problems on their own and then I tried to get any clues to see if they might be working in researchers are plain of study dozens of patients and medical centers in this country and in Canada and Europe it could take months and maybe even years to know how well it's working can I talk to Victoria about that she says she knows the risks and that is a very early study but she can't help but hope that help Sir I feel like the way everything happened he was kinda fate enough feel special food being the first

Christopher
A Brief History of Sickle Cell Disease

The Pulse

02:09 min | 2 years ago

A Brief History of Sickle Cell Disease

"Story is about a disease that could have been mostly wiped out. But it wasn't. It's a story about a public health campaign gone wrong, sickle cell disease is an inherited blood disorder blood cells bend into a sickle shape, which causes blockages and starves tissue of oxygen this illness affects people of African descent as well as Mediterranean and south Indian peoples. The American government took aim at sickle cell in the nineteen. Seventies jets Lehman reports on how public health missed the Mark. It's March nineteen seventy-two. Bobby Seale speaking in a crowded gym. And we're not saying that. Vava programs are necessarily revolutionary. He's the co founder of the Black Panther party, a radical African American civil rights organization founded in California survivor programs to and institutions by which we unify people around Abana reads sickle cell, anemia testing black community survival conference a blood test shows if you have the sickle cell trait or not. Trait carriers usually don't have any symptoms. But if two people who have the trait have children their kids may develop sickle cell disease, which can be fatal the Black Panthers had been calling attention to sickle cell for years. And it looked like the government had finally started to listen congress had approved a fivefold increase in sickle cell research. Funding, president Nixon made fighting the disease a priority. America has long been the wealthiest nation in the world. Now, it is time we became the healthiest nation in the work sickle cell testing had been possible for decades. Now there was funding as wills a mandate from the leader of the free world. We were going to stop sickle cell in America once and for all. Only we didn't. This is a young girl in her bed. Tears streaming. She's in sickle cell crisis.

Sickle Cell Trait Black Panther Party Bobby Seale American Government America Blood Disorder President Nixon Mediterranean Lehman Congress Abana California
What are the potential health benefits of gene editing?

FT News

13:46 min | 2 years ago

What are the potential health benefits of gene editing?

"Last year. A Chinese scientists shocked the world by disclosing hit created the world's first gene, edited babies, and John Ahuja talks to Robin level badge a developmental biologist geneticist about the controversy and about the potential for easy to use gene editing, tools such as crisper Cas nine to revolutionize diagnostics, drug discovery and the treatment of disease. Everybody has become very familiar with the story of the gene edited babies that came out from China last year. So these were apparently, the world's first genetic babies created it really triggered a scandal. Do we know what's happened to the scientist and author the twin baby girls that have been born? We know nothing new particularly drunk you. Hey, all vote voters JK is currently in Shenzhen where here's university is. I think he's in a an apartment owned by the university and the stories in the Chinese press that the gods. What we don't know who those gods are whether the university outs police, whoever they are. We don't know whether he's under house arrest or whether he's free to move around. He actually emailed me and said, he's all. Fine. So the impression is I think he's trying to give me that he's not under arrest. So the goals may be that to protect him because I know he was receiving threats during the become fronts that we had last hole. I can say he's fine. About the welfare of the twin girls. Lulu a Nana be concern. We know nothing about that. Jake himself was very keen to stress that that privacy should be maintained that they should be protected and density, not known on. We'll this likewise their parents. There is an investigation being launched in China by the ministries of sciences on administrative health. They presumably will want to find out whether what he's claimed to have done is actually the case. And so they would need to take DNA samples from the two babies, and the parents, but I hope they do it in a sensitive way. These are just two little babies. They should be cheated just as that and not subject to anything bad at all. They have novel mutations in this, gene L five, which is known in some cases to confer protection, it mutations in that gene can confer protection against HIV. And that was the rationale behind the work. But Jake I did was to try and make these children immune if you like to HIV because having trophy in China of even being a member of a family where one of the family members house HIV as in this case, it's cheap very badly. Families the stigmatized the children wouldn't be allowed to play with other children simply because of that for example. So that also sized about another reason why the density needs to be protected because of that situation too. But we hope is that they will teach it to normal little girls. And that's it. They have mutations in the gene that novel mutations because of the way he did the dean of editing quite poorly. We have no idea what those mutations will do but all of us have mutations. Every new generation that forty two eighteen you mutations in genome. So just treat them like normal children. Keep a watching eye on them. See what happens say watch this space on the genus babies story. But of course, may scientists involved in genome editing, as you prefer to call it on not involved in trying to change the course of human evily Shen by creating these genetic mutations or changes that will then be passed on down the family line, as I understand it the real excitement for genome editing is in the lab working with adult patients, perhaps with single gene disorders also excitement with drug discovery and so on so let's talk about that. Because I know that you were involved as developmental biologist in the aren- decide of things does your work focused on the creek. We have a number protecting many projects, but let me decide about one of them jeans have to pulse to them. They have the part that encodes the protein is going to do its job for it. But it also has a part but controls when and where that gene is active so normal genes, right? Even all cells over time, they all cell type specific, and they can be. Stage of development specific they can be on the active of particular time in the embryo or any adult we've been using the genome editing methods to try and understand bisect light. This regulatory region for some jeans and one in particular, cold sulks nine so soaks nine is gene that has many roles in developing embryo, but one we'd been working on for a long time as its role in sex determination. So whether you become male-female soaks nine is really critical to give rise to the development of testes particular cell type was critical for making tested so mutations in soak nine can lead to sex reversal to give ex wife female development instead of male development. But it's a really complex gene is active in many different cells. And it turns out that it has an enormous regulatory region, which is really complex and we wanted to try and dissect that say you have specific sequences you can refer to enhance or sequences where other proteins transcription factors in Toronto. With those. To tell the gene to be active or silent. And so we had fun using variety of methods number of candidate regimes run home of for soaks nine in the process of making testes we had about thirty two different candidates. We started with and we use number methods to try and reduce that number down. We came down to four but looked like they were very promising, and we use genome editing methods to inactivate each of those in enhances and turn to basically delete each of those regions of DNA intern unfound one of them, which is located very long distance away from the protein, coding Potter, gene itself. Some six hundred fifty thousand base pairs away which is big distance. When we did he should that particular in Holzer it inactivates, the, gene. So we got X Y females. So even though you have this really complex regulatory region. Spread over many millions of base pairs and stuffed full of different regulatory regions. Turns out, but just one was essential for expression in the developing gonads said that they can rise to test. What's the next female? They are crumbs Emily mail voicemails scream saying about they've developed as a female in this case in the embryos. They are indistinguishable from normal females stabbed genotype female genitally. This is women. They have the mice. Not women they develop his females in humans, of course. Yes. You would also have cases where you have extra female development. So they look female, but chromosome Yuda male. They will be infertile because the germs house of the eggs in the next female that do very well, they get lost early on. So they will be infertile. But otherwise, they look and generally behave and all female month thing, I should ask you to do been is to explain very briefly. What's crispy cast Moines editing, as can you tell us how the crisper cast nine genome editing system works, very briefly, please, okay. It relies on making breaks in the DNA usually a double strand break in the day, and to do that you have an enzyme called cows nine, but you have to have a way of getting not enzyme to the right place in the genome. And that's the guide are a component. So the RA you design it part of the irony is designed to be complementary. So to match up with the DNA sequence that you want to cut the other part of the RA guide are on a has a sequence of interacts with the cows nine enzyme protein. So when you have both together, and you crisper is not linking crisper yours. Crisper is a shorthand term for the guide our nights for the irony. The name crisper comes from origin. Bacteria is not really relevant to. Use to the guy Rene takes the Cas nine enzyme to the right part of the genome. The enzyme an cups, the DNA and processes that occur in also to repair broken deny then jump in and try and repair it now in the simplest form of technology. You just making a break in the DNA actually, Germany making a mutation in a gene, you have a process called known homogeneous end joining horrible term, but it basically just tries to stick the broken ends DNA bound together again, but often you have a little mistake made and that can be sufficient to an activator, gene. This is incredibly useful in the lab for studying gene functions say rapid and easy. Now, if you want to make them more precise alteration in the genome sequence DNA sequence on that can be anything from a single letter up too, many many thousands of letters of code then you have to use a different DNA repair mechanism cold Hamasi directed repair. This requires a third. Component to be introduced to the time time, which we confined to his DNA template. So this has to pulse. Each end. You have a bit of DNA, but complementaries of that's the same as the gene in which you want to talk it. And then in the middle. You have a sequence that you want to replace the one that's in our ready with and if you have those three components together. Now, the cows names aren't the guide our day on the DNA template the direction repair mechanism will not work, and it will substitute. What was in old template into the gene it is quite an incredible technology in it? There's a third method which I wanted to tell you about which is really exciting, which is cold base editing. Now, this allows you to change one base pair one letter if you like in the code, but it works without making a double strand, cutting the DNA, it changes chemically if you like one letter to another then it's partner also then has to change. Change. Otherwise, you have a little loop in the DNA, and that relies on a different type of DNA repair maximum to do a little bit. But given that about half of single, gene disorders or calls. In fact, by single base pair changes mutations in the DNA, this new methods looks really promising because it doesn't come with the baggage that some of the other methods have of creating unwanted mutations. So you get just what you want. So it could well be about safer method at least when you're dealing with single based mutation. Tell me how genome editing is being used in the lab, and perhaps with patients, it's really exploded. In basic research. The use of genome entertain particular the crisp cows nine methodology, it's very easy to use. That's one reason relatively inexpensive to use. But it's just so powerful and it's a rapid. So we've had ways of altering genes in cells in coach. Uh-huh. Or in model animals that we use for research like mice methods to alter genes in many years for decades, but they will always very inefficient very slow to make a mouse carrying specific mutation in a particular, gene, it really used to take well over a year to do that we cannot do it in the motto of a couple of months. So it rapidly speeds up the way that we can do research to study the role of specific genes or pulse of genes during development in my case or for physiology over brain function, or in cancer, it's really speeded up things enormously and made it cheaper in terms of things like the possibility of using genome editing to treat patients who already have a genetic disease or council something like this again, that's really looking incredibly exciting. We've had some somebody, gene therapy. Also for decades, just explain what semitic therapies somatic cells are. Basically any sewn in the body apart from the jumps health germ cells being themselves who are going to give rise to sperm or eggs that would allow any genetic changed repulsed on to subsequent generations. So a typical somebody will be a skin owl muscle tell or bone cell brain zone. So there are whole ranger Nettie disease is that can compromise. The ability of particular cells to function or the politics function things that the blood'll sickle cell disease will be catalyse Mia, you have disease like cystic fibrosis or muscular dystrophy affect lungs muscles opticians, in many cases, these G two single mutations Chinga, gene. It's affected by small mutation affecting that, gene. So the genome editing methods can be used to correct, gene defects, or in some cases, find ways around of getting another gene to become active to replace the gene that's not working. There are now probably around twenty or so clinical trials that had been launched. Using the female amenity methods to try and treat individuals who have think disease of this soaks. I'm single, gene defects.

China Scientist Jake Shenzhen John Ahuja Toronto Lulu Robin Holzer Emily Intern MIA Potter Rene Germany Chinga Partner
Multi-gene test may find risk of heart disease and more

24 Hour News

01:37 min | 2 years ago

Multi-gene test may find risk of heart disease and more

"Researchers have developed a, new way to analyze genetic data AP's Jackie Quinn reports it may one day help, identify people at high risk of combinations like heart disease and diabetes genetic testing right now mostly focuses on rare mutations like ones. That can cause breast, cancer sickle cell disease and cystic fibrosis now research With

Musk Caspian Sea Tesla Saudi Arabia Soviet Union Jackie Quinn Kazakhstan Azerbaijan Russia Stan Iran Twenty Dollars One Day
"sickle cell disease" Discussed on KQED Radio

KQED Radio

02:01 min | 3 years ago

"sickle cell disease" Discussed on KQED Radio

"Sickle cell disease and is the founder of destiny despite diagnosis brad asked a good question dr lands krahn sickle cell disease versus sickle cell anemia i think growing up i heard a lot about sickle cell anemia but what's the difference are the the same thing or is one kind of a components of the other it's knows the now diseases the term we use to encompass all different types of sickle cell disease and there are other mutations of hemoglobin such as he go c or a phthalates cmea mutation that when coupled with an s a sicle mutation causes sickle cell disease but the most severe form of sickle cell disease is what we call sickle cell anemia where you inherit an as from mom and an as from dad and really only make him a globe in apps and also people who have a another very rare mutation called beta zero thousand a'mia which when coupled with as he really only make sickle hemoglobin if you know what kind of sickle cell disease you have i do i have ss disease and i when i do education and workshops i make a point for people to know if they don't know and they should find out uh because it's very important and how you treat your disease condolences to george m who wrote in saying that he lost a friend to this disease also noted about it being a mutation to protect one from malaria jenny gold there was just a report a few days ago about pop star jordan sparks who lost four loved ones back to back to back to back including one who died of sickle cell i believe it was her stepsister briana jackson fries who died at age sixteen about a week ago of of sickle cell it seems like that's kind of when i wonder if in your research you find that's kind of when it hits home that people don't really think much about it until someone becomes yoga mortally hospitalized or someone passes way it's just a terrible story that someone died at sixteen generally pediatric care for sickle cell disease.

founder brad malaria george m briana jackson