Long-term survival and cardiac efficacy of a gene therapy for Duchenne muscular dystrophy

A new study published in the peer-reviewed journal Human Gene Therapy evaluated the long-term survival and cardiac efficacy of the gene therapy delandistrogene moxeparvovec in a rat model of Duchenne muscular dystrophy (DMD). Click here to read the article now.

Delandistrogene moxeparvovec uses an adeno-associated viral (AAV) vector to deliver a micro-dystrophin transgene to skeletal and cardiac muscle.

Rachel Potter and coauthors from Sarepta Therapeutics injected DMD rats with delandistrogene moxeparvovec and then assessed ambulation and various metrics of cardiac disease at 12, 24, and 52 weeks post-treatment.

Treatment with the gene therapy extended the median survival of DMD rats to >25 months, compared to the 13-month median lifespan for the control group of DMD rats. Compared to the control rats, delandistrogene moxeparvovec therapy elicited statistically significant improvements across cardiac parameters, approaching wild-type values, with additional benefits in mobility.

The investigators reported that “transgene expression was maintained up to >25 months and micro-dystrophin expression was broadly distributed across skeletal and cardiac muscle.”

“This is encouraging data that the clinically approved gene therapy delandistrogene moxeparvovec produces functional improvements in cardiac muscle to combat DMD-induced cardiomyopathy,” says Managing Editor of Human Gene Therapy Thomas Gallagher, PhD, from the University of Massachusetts Chan Medical School.

About the Journal
Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy and eight other international gene therapy societies, was the first peer-reviewed journal in the field and provides all-inclusive access to the critical pillars of human gene therapy: research, methods, and clinical applications. The Journal is led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, and an esteemed international editorial board. Human Gene Therapy is available in print and online. Complete tables of contents and a sample issue are available on the Human Gene Therapy website.

About the Publisher
Mary Ann Liebert, Inc. is a global media company dedicated to creating, curating, and delivering impactful peer-reviewed research and authoritative content services to advance the fields of biotechnology and the life sciences, specialized clinical medicine, and public health and policy. For complete information, please visit the Mary Ann Liebert, Inc. website.