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CNS-ICNA 2020

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Novel gene therapy for DMD induces expression of full-length dystrophin

A novel gene therapy may induce full-length dystrophin expression in patients with Duchenne muscular dystrophy (DMD), according to preliminary results from an ongoing clinical trial.

The therapy is an exon-skipping therapy targeting exon 2, the most commonly duplicated exon in the DMD gene, according to Dr Megan Waldrop of Nationwide Children’s Hospital in Columbus, Ohio, USA.

In a first-in-human trial, Dr Waldrop and colleagues assessed the effects of the gene therapy in patients with DMD. Treatment included intravenous administration of a viral vector (scAAV9.U7-ACCA) containing small nuclear RNA constructs that target the splice donor and acceptor domains of exon 2.

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