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Gene therapy in monogenic congenital myopathies
- Guan, Xuan, Goddard, Melissa A., Mack, David L., Childers, Martin K.
- Methods 2016 v.99 pp. 91-98
- animal models, dogs, gene therapy, genes, muscular diseases, patients
- Current treatment options for patients with monogenetic congenital myopathies (MCM) ameliorate the symptoms of the disorder without resolving the underlying cause. However, gene therapies are being developed where the mutated or deficient gene target is replaced. Preclinical findings in animal models appear promising, as illustrated by gene replacement for X-linked myotubular myopathy (XLMTM) in canine and murine models. Prospective applications and approaches to gene replacement therapy, using these disorders as examples, are discussed in this review.