Fixing a Genetic Flaw22 Mar
A novel form of antisense therapy known as exon skipping proves
successful in treating a variant of muscular dystrophy in dogs in the
lab.
2 Responses to “Fixing a Genetic Flaw”
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Now that’s clever! Not replacing the missing DNA, but fixing what good DNA is there to make the gene work as well as it can.
Yeah, pretty cool, huh? Antisense approaches are still expensive and difficult to deliver – these poor dogs had to put up with constant injections – but where those issues can be surmounted they can solve problems very hard to tackle with traditional drugs.
We miss you, btw – when are you coming to visit San Diego?