New Discovery Enables Gene Therapy for Muscular Dystrophies, Other Disorders

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From the University of Rochester Medical Center Newsroom:

RNA-based technology facilitates effective use for difficult-to-treat, large-gene diseases

Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is a big problem: size. The genes that are dysfunctional in muscular dystrophies are often extremely large, and current delivery methods can’t courier such substantial genetic loads into the body. A new technology, dubbed “StitchR,” surmounts this obstacle by delivering two halves of a gene separately; once in a cell, both DNA segments generate messenger RNAs (mRNAs) that join seamlessly together to restore expression of a protein that is missing or inactive in disease.

Study authors from the Anderson Lab

Published in the journal Science, StitchR—short for “stitch RNA”—restored expression of large therapeutic muscle proteins to normal levels in two different animal models of muscular dystrophy. StitchR enabled expression of the protein Dysferlin, which is lacking in individuals with limb girdle muscular dystrophy type 2B/R2, as well as the protein Dystrophin, which is absent in patients with Duchenne muscular dystrophy. (click here to keep reading)

Author: Emily Boynton