Gene therapy leads to robust improvements in animal model of fatal muscle disease

Preclinical studies show that gene therapy can strengthen muscles and lengthen lives in animal models of a fatal congenital disease in children, X-linked myotubular myopathy. The findings demonstrate the clinical feasibility of future trials for this devastating disease. Children born with the condition have floppy muscles and breathing difficulty, and may need life support. Most die in childhood. The effectiveness of this process was tested in mice and dogs with engineered adenovirus vector carrying a gene to replace the mutation.


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