Systemic γ-sarcoglycan AAV gene transfer results in dose-dependent correction of muscle deficits in the LGMD 2C/R5 mouse model

Limb-girdle muscular dystrophy (LGMD) type 2C/R5 results from mutations in the γ-sarcoglycan (SGCG) gene and is characterized by muscle weakness and progressive wasting. Loss of functional γ-sarcoglycan protein in the dystrophin-associated protein complex destabilizes the sarcolemma, leading to even...

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Detalles Bibliográficos
Autores principales: Seo, Young-Eun, Baine, Stephen H., Kempton, Amber N., Rogers, Oliver C., Lewis, Sarah, Adegboye, Kaitlin, Haile, Alex, Griffin, Danielle A., Peterson, Ellyn L., Pozsgai, Eric R., Potter, Rachael A., Rodino-Klapac, Louise R.
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/PMC9929442/
https://www.ncbi.nlm.nih.gov/pubmed/36816759
http://dx.doi.org/10.1016/j.omtm.2023.01.004

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9929442/
https://www.ncbi.nlm.nih.gov/pubmed/36816759
http://dx.doi.org/10.1016/j.omtm.2023.01.004

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