Joe Mcconnell, Theodore Urnov, Patrick Doherty discussed on Morning Edition


CRISPR into patients bloodstreams to try and help them and it appears to have worked. NPR health correspondent Rob Stein has more Patrick Doherty had always been really active, trekking the Himalayas, hiking trails in Spain, but he suddenly started feeling pins and needles in his fingers and toes, and his feet got cold. And then one day, he noticed he was running out of breath just walking his dog in County Donegal in Ireland, where he lives and kind of getting a bit breathless going up. Uh, a small, l said here in Donegal, which which There are many. So I realized something was something was wrong. Something was wrong. Turns out he had inherited a rare but devastating disease that killed his dad, known as amyloid doses. A deformed protein was damaging parts of his body like nerves in his fingers and toes and his heart. He had watched two uncles and neighbours also die awful deaths from amyloidosis. It's just dreadful. So Doherty, who's 65 was thrilled when he found out that doctors were testing. A new way to treat amyloidosis using CRISPR, which allows scientists to make very precise changes in DNA. I thought fantastic, I couldn't believe my look crisper has already started helping patients suffering from the blood disorders, sickle cell disease and beta thalassemia, and doctors are trying to use it to treat cancer and restore vision. Two people blinded by a rare genetic disorder, but those experiments involved taking cells out of the body. Editing them in the lab and infusing them back in or injecting CRISPR directly into cells that need fixing. The study. Doherty volunteered for is the first in which doctors are simply infusing the gene editor and letting it find its own way to the right gene in the right place in this case, a gene and cells in the liver Pumping out the destructive protein. John Leonard is the CEO of Antalya Therapeutics, the company sponsoring the study. This is the first example in which CRISPR CAS nine is injected directly into the bloodstream, in other words, systemic administration where we use as a way to reach a tissue that's far away from the site of injection and very specifically edit particular disease causing genes, and it looks like it worked. Doctors infuse billions of micro This topic structures known as nanoparticles into Doherty and five other volunteers. Each nanoparticle carried the genetic code for the CRISPR gene editor. The nanoparticles made their way to deliver And unleashed an army of crisp routine editors, which honed in on the target gene to disable it. Within weeks, the levels of bad protein causing the disease plummeted. It's amazing. It's uh, you know, it's it's phenomenon, It really is excited. Dr. Julian Gilmore is leading the study at the University College London. This has the potential to Completely revolutionized the outcome for these patients who have lived with this disease in their family for many generations, you know, it's decimated some families that I'm that I've been looking after. So now this is this is amazing. The patients will have to be followed much longer, and many more patients will have to be treated to make sure the treatments as safe as it looks and to determine how much it's helping patients. But Gilmour says the approach could help those struck by a far more common version of the Disease and provides the first good evidence that CRISPR could be used like this to treat many other, much more common diseases for which taking cells out of the body or directly injecting CRISPR isn't realistic, like heart disease, muscular dystrophy and maybe even brain disorders like Alzheimer's. This is a wonderful day for the future of gene editing as a medicine. Theodore Urnov is a professor of genetics at the University of California, Berkeley. What's striking about These new data is not merely the evidence of safety and efficacy but the avenues or highways. It opens up for us in terms of traveling to new destinations in the world of genomic medicine, he says. It's a new world. We as a species are watching this remarkable new show called R gene edited future. For his part, Patrick Doherty is thrilled. I feel I feel fantastic, feel fantastic. He started feeling better within weeks of the treatment. Definitely notice an improvement in terms of mobility, My my, my heart. They move in a boat. I'm walking again. I do 15 35,000 steps a day and weekend I got again. He'll walk in with a dog. Doherty's looking forward to the future again. I'm really thankful for it really thankful for, And he hopes CRISPR will help some of his friends and relatives avoid the terrible fate. His father, uncles and friends suffered. Rob Stein. NPR news This is NPR news over to Joe McConnell at the traffic desk at 7 19 on KQED Jail Reporting of art delay. The 10 minute delay on the various align heading in the Richmond and Daly City directions because of some police activity at Hayward and South Hayward stations on the freeways. It's been pretty uneventful. Just a few bender a fender benders of South Adedy before autumn, a park where it looks like that one has cleared still heavy from Thornton, but picking up up to 25 30 Miles an hour now. Did the South I 2 to 37. There might be some metal debris in the right lane..

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