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Development of a high-throughput screen to identify small molecule enhancers of sarcospan for the treatment of Duchenne muscular dystrophy

Abstract Background Duchenne muscular dystrophy (DMD) is caused by loss of sarcolemma connection to the extracellular matrix. Transgenic overexpression of the transmembrane protein sarcospan (SSPN) in the DMD mdx mouse model significantly reduces disease pathology by restoring membrane adhesion. Ide...

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Autori principali: Cynthia Shu, Ariana N. Kaxon-Rupp, Judd R. Collado, Robert Damoiseaux, Rachelle H. Crosbie
Natura: Artigo
Lingua:Inglês
Pubblicazione: BMC 2019-12-01
Serie:Skeletal Muscle
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Accesso online:https://doi.org/10.1186/s13395-019-0218-x
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