Gene Editing CRISPR/Cas9 Therapy for Duchenne Muscular Dystrophy

Dr. Eric Olson describes a gene editing therapeutic approach to correct underlying genetic mutation in Duchenne muscular dystrophy.

Exonics Therapeutics, Inc., is a newly formed biotechnology company focused on developing gene editing technologies like CRISPR/Cas9 to permanently correct a majority of mutations causing Duchenne muscular dystrophy and other neuromuscular diseases. CureDuchenne Ventures, LLC, a subsidiary of the nonprofit CureDuchenne, provided seed funding to allow Exonics to advance the preclinical research of its scientific founder and chief science advisor Eric Olson, PhD.

Dr. Olson’s laboratory has demonstrated the ability to use adeno-associated virus (AAV) to deliver a payload based on CRISPR/Cas9 technology that can identify and correct exon mutations that prevent the production of dystrophin, a protein that helps stabilize and protect muscle fibers. Dystrophin is the key protein missing in boys with Duchenne, and published preclinical data suggest that this approach has the potential to permanently treat up to 80 percent of children suffering from the disease.

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