Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit

Mutations of the DYSF gene leading to reduced dysferlin protein level causes limb girdle muscular dystrophy type 2B (LGMD2B). Dysferlin facilitates sarcolemmal membrane repair in healthy myofibers, thus its deficit compromises myofiber repair and leads to chronic muscle inflammation. An experimental...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Sreetama, Sen Chandra, Chandra, Goutam, Van der Meulen, Jack H., Ahmad, Mohammad Mahad, Suzuki, Peter, Bhuvanendran, Shivaprasad, Nagaraju, Kanneboyina, Hoffman, Eric P., Jaiswal, Jyoti K.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2018
Assuntos:
Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6127637/
https://ncbi.nlm.nih.gov/pubmed/30166241
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.07.021
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