How Gene Therapy Helped Conner Run

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Mattie. SAFAI NPR science correspondent. John Hamilton Hi John Hi Mary so John, where would you like to begin I? Think we should start with the scientist. Okay. Let's do it. Okay. So obviously many many scientists have worked to understand this disorder. But today we're gonNA focus on Jude Samal ski back in Nineteen eighty-four and I'll ski was still a graduate student at the University of Florida and he was part of this team that cloned a virus called A V. and those are group of viruses that can infect people but they don't cause diseases. Yeah. I remember I learning about this in Grad School John that discovery was a big deal because basically we can turn these viruses in tools and and that's because viruses on their own are pros at getting into ourselves and getting up close and personal with our DNA, which is exactly where you need to get to treat a lot of genetic disorders at. Their source exactly, and he was one of the scientists who figure that out. So as you these viruses have just revolutionized gene therapy right and after some Oh ski and his team Clone Davie, they wanted to try to use the virus to treat descend muscular dystrophy. That's the genetic disorder you were talking about earlier. Got It. So a lot of these therapies work by kind of targeting gene or genes that are the root of a disorder. So what's The deal with to Sheng muscular dystrophy John Kids who have Sharon. Lack a functional version of gene called D. M. D., and this gene makes a protein called destroyed often that helps muscles stay healthy. Got It. Okay. The idea is if the problem is that someone lack a working gene, you could just give them a working copy of that gene and what's the most wanted to do was packed some of the genetic code from a disrobing gene inside. Right and then once the virus got into the body, it would infect muscle cells, and then that faulty code is replaced with a functional version. Right? smokey says a Aviv, this harmless virus would work. Station service it's a molecular Fedex truck. Carries a genetic payload and it's delivering to its target right but it turns out bring a gene is a little bit harder. Then delivering a package and destroyed gene is especially challenging. One reason is it's is the a the virus are Fedex truck is incredibly tiny even among viruses. It's so small. You need an electron microscope just to see it, and then you have the destroyed gene, which is huge. It's the largest known human gene it contains about five. Hundred Times more genetic code than a so fitting that specific gene into that specific virus would be like trying to get a football stadium into a fedex truck something like that. Yeah, and most he has some other challenges to One is that do sheng affects billions of muscle cells all over the body. So this a delivery truck would have to be programmed to find all of these cells recognize them, and then infect them with this new genetic code. Yeah and some spent fifteen years tackling these challenges he was going along you is making progress he said, but it was coming one small step at a time. This is very challenging. It was mount ever said the gene therapy community in each one of these steps was setting up base camp, but then in nineteen, ninety, nine so mulcahy's work for that matter all gene therapy research pretty much came to a stop. The reason was that a teenager named Jesse. Gelsinger had died in the gene therapy experiment, right? I. Mean I. Remember Learning about that in graduate school in genetics. It was horrible. It was really sad the experiment he was part of had nothing to do with muscular dystrophy or the virus nothing to do with some all skis work, but it didn't matter right gene therapy trials were postponed or abandoned investors disappeared and so did research funding it stopped everything everyone got supercautious everyone except the muscular dystrophy association. The Jerry Lewis Telethon people they continue to push for the advancement of gene

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