DNAJB6 isoform specific knockdown: Therapeutic potential for limb girdle muscular dystrophy D1

Dominant missense mutations in DNAJB6, a co-chaperone of HSP70, cause limb girdle muscular dystrophy (LGMD) D1. No treatments are currently available. Two isoforms exist, DNAJB6a and DNAJB6b, each with distinct localizations in muscle. Mutations reside in both isoforms, yet evidence suggests that DN...

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Detalles Bibliográficos
Autores principales: Findlay, Andrew R., Paing, May M., Daw, Jil A., Haller, Meade, Bengoechea, Rocio, Pittman, Sara K., Li, Shan, Wang, Feng, Miller, Timothy M., True, Heather L., Chou, Tsui-Fen, Weihl, Conrad C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10280091/
https://www.ncbi.nlm.nih.gov/pubmed/37346979
http://dx.doi.org/10.1016/j.omtn.2023.05.017

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