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How might gene therapy be used to treat muscular dystrophies?

3 doctors weighed in
Dr. Kathryn Mosher
Physical & Rehabilitation Medicine
1 doctor agrees

In brief: In hopes to cure

Gene therapy is targeted at replacing the damaged segments of dna with more normal copies so that the body can produce adequate dystrophin--the protein missing in duchenne.
So far no one has found a method that successfully delivers the gene, largely because it is very big. But there is still a lot of work in this area being done.

In brief: In hopes to cure

Gene therapy is targeted at replacing the damaged segments of dna with more normal copies so that the body can produce adequate dystrophin--the protein missing in duchenne.
So far no one has found a method that successfully delivers the gene, largely because it is very big. But there is still a lot of work in this area being done.
Dr. Kathryn Mosher
Dr. Kathryn Mosher
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1 comment
Dr. Ricardo Madrid
That was the problem initially, but nowadays exon skipping for reading frame restoration is being used in Duchenne patients. The best results so far have resulted in partial expression of hte dystrophin protein in muscle fibers, like reverting a Duchenne for a Becker phenotype.
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