Large1 gene transfer in older myd mice with severe muscular dystrophy restores muscle function and greatly improves survival

Muscular dystrophy is a progressive and ultimately lethal neuromuscular disease. Although gene editing and gene transfer hold great promise as therapies when administered before the onset of severe clinical symptoms, it is unclear whether these strategies can restore muscle function and improve surv...

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Detalles Bibliográficos
Autores principales: Yonekawa, Takahiro, Rauckhorst, Adam J., El-Hattab, Sara, Cuellar, Marco A., Venzke, David, Anderson, Mary E., Okuma, Hidehiko, Pewa, Alvin D., Taylor, Eric B., Campbell, Kevin P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Association for the Advancement of Science 2022
Materias:
Biomedicine and Life Sciences
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9132445/
https://www.ncbi.nlm.nih.gov/pubmed/35613260
http://dx.doi.org/10.1126/sciadv.abn0379

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9132445/
https://www.ncbi.nlm.nih.gov/pubmed/35613260
http://dx.doi.org/10.1126/sciadv.abn0379

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