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Gene delivery goes global

For gene therapy of muscular dystrophies, all of the skeletal muscles in the body must be transduced—a tough challenge. An approach that permeabilizes blood vessels using VEGF, helping low doses of a parvovirus-based gene therapy vector sneak across the vessel wall, now solves this problem in mice (pages 828–834).

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Figure 1: Delivery of a therapeutic gene to the skeletal muscles and heart of an adult animal with muscular dystrophy.

Paul Gregorevic

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Thomas, C. Gene delivery goes global. Nat Med 10, 782–783 (2004). https://doi.org/10.1038/nm0804-782

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