Sarepta Therapeutics Enters into License Agreement with Nationwide Children’s Hospital for Galgt2 Gene Therapy Program

The experimental program explores the potential surrogate gene therapy approach to Duchenne muscular dystrophy. As a “surrogate gene therapy approach”, the gene therapy looks to induce genes that make proteins that can perform a similar function as dystrophin, with the goal of producing a muscle cell that can function normally even when dystrophin is absent. It has the potential to be used broadly in several muscular dystrophies

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