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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Vydáno v:Mol Ther
Hlavní autoři: 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.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Original Article
On-line přístup: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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