Article
Dynamin-2 reduction rescues the skeletal myopathy of a SPEG-deficient mouse model.
JCI insight - 8 Aug 2022
Li Qifei, Lin Jasmine, Widrick Jeffrey J, Luo Shiyu, Li Gu, Zhang Yuanfan, Laporte Jocelyn, Perrella Mark A, Liu Xiaoli, Agrawal Pankaj B
Abstract excerpt
Striated preferentially expressed protein kinase (SPEG), a myosin light chain kinase, is mutated in centronuclear myopathy (CNM) and/or dilated cardiomyopathy. No precise therapies are available for this disorder, and gene replacement therapy is not a feasible option due to the large size of SPEG. We evaluated the potential of dynamin-2 (DNM2) reduction as a potential therapeutic strategy because it has been...
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