Gene therapy in monogenic congenital myopathies

Xuan Guan , Melissa Goddard (1) , David Mack , Martin K. Childers (2)

1 INTEGRARE - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares
2 Wake Forest University

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.

Keywords: Animal models; Gene therapy; Muscle disease; Muscular dystrophy; Myopathy

Domains: Life Sciences [q-bio]

Xuan Guan, Melissa Goddard, David Mack, Martin K. Childers. Gene therapy in monogenic congenital myopathies. Methods, 2016, 99, pp.91-98. ⟨10.1016/j.ymeth.2015.10.004⟩. ⟨hal-02180076⟩