Repression of phosphatidylinositol transfer protein Î± ameliorates the pathology of Duchenne muscular dystrophy.

Proc. Natl. Acad. Sci. U.S.A.2017 Jun 06;114(23):6080-6085. Epub 2017 May 22

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as a disease modifier that accords benefits to the abnormal signaling, morphology, and function of dystrophic skeletal muscle, and may be a target for DMD and related neuromuscular diseases.

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Repression of phosphatidylinositol transfer protein Î± ameliorates the pathology of Duchenne muscular dystrophy.

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