Discovery enables effective use of gene therapy for muscular dystrophies and other 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.

This article was originally published on MedicalXpress.com

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