Article
Repression of phosphatidylinositol transfer protein α ameliorates the pathology of Duchenne muscular dystrophy.
Proceedings of the National Academy of Sciences of the United States of America - 6 Jun 2017
Vieira Natassia M, Spinazzola Janelle M, Alexander Matthew S, Moreira Yuri B, Kawahara Genri, Gibbs Devin E, Mead Lillian C, Verjovski-Almeida Sergio, Zatz Mayana, Kunkel Louis M
Abstract excerpt
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by X-linked inherited mutations in the DYSTROPHIN (DMD) gene. Absence of dystrophin protein from the sarcolemma causes severe muscle degeneration, fibrosis, and inflammation, ultimately leading to cardiorespiratory failure and premature death. Although there are several promising strategies under investigation to restore dystrophin...
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