Secreted acid sphingomyelinase as a potential gene therapy for limb girdle muscular dystrophy 2B

Efficient sarcolemmal repair is required for muscle cell survival, with deficits in this process leading to muscle degeneration. Lack of the sarcolemmal protein dysferlin impairs sarcolemmal repair by reducing secretion of the enzyme acid sphingomyelinase (ASM), and causes limb girdle muscular dystr...

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Detalles Bibliográficos
Autores principales: Bittel, Daniel C., Sreetama, Sen Chandra, Chandra, Goutam, Ziegler, Robin, Nagaraju, Kanneboyina, Van der Meulen, Jack H., Jaiswal, Jyoti K.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8718136/
https://www.ncbi.nlm.nih.gov/pubmed/34981776
http://dx.doi.org/10.1172/JCI141295

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