Gene Therapy

Gene Therapy Shows Great Promise in Treatment of DMD

Gene therapy is rapidly advancing the ability to treat Duchenne at a genetic level. DMD is caused in part by a mutation of the gene which produces dystrophin, the protein responsible for building healthy muscle. Researchers in Canada and Japan have recently found a way to alter the genetic code, known as exon skipping, in order to signal the body to produce dystrophin. By skipping the mutated genes, a percentage of DMD patients are able to produce the dystrophin they lack. While the process is still in the trial phase, this discovery holds the promise of a ground-breaking viable therapeutic option for those suffering from Duchenne.


Canadian Researchers Discover CRISPR Gene Editing Helps Heal Mice With Muscular Dystrophy

Japan Researchers Prove Long-term Efficacy and Safety of Exon-Skipping Therapy

Gene Therapy


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