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Duchenne muscular dystrophy (DMD) is an X-linked recessive disorder caused by defects in the dystrophin gene. Patients exhibit progressive muscle degeneration, generally begin using a wheelchair before 11 years of age, and die from respiratory or cardiac failure by their early to mid-twenties. Currently no treatment is available for this disease that affects 1 in 3500 newborn males worldwide. As a result, intensive efforts are under way to develop methods to treat DMD by gene therapy. This Commentary will present some of the successes of this research and identify a few of the most challenging obstacles to be overcome before effective treatment is available.

Success in treating any disorder by gene therapy hinges on a thorough understanding of the biological parameters of the disease process and the tissues involved. Much of this groundwork has been laid for DMD, and this research effort can serve as a paradigm for the assault