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Efficacy and muscle safety assessment of fukutin-related protein gene therapy

Limb-girdle muscular dystrophy type R9 (LGMDR9) is a muscle-wasting disease that begins in the hip and shoulder regions of the body. This disease is caused by mutations in fukutin-related protein (FKRP), a glycosyltransferase critical for maintaining muscle cell integrity. Here we investigated poten...

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Detalles Bibliográficos
Autores principales:	Benasutti, Halli, Maricelli, Joseph W., Seto, Jane, Hall, John, Halbert, Christine, Wicki, Jacqueline, Heusgen, Lydia, Purvis, Nicholas, Regnier, Michael, Lin, David C., Rodgers, Buel D., Chamberlain, Jeffrey S.
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/PMC10285450/ https://www.ncbi.nlm.nih.gov/pubmed/37361354 http://dx.doi.org/10.1016/j.omtm.2023.05.022

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