Preclinical Systemic Delivery of Adeno-Associated α-Sarcoglycan Gene Transfer for Limb-Girdle Muscular Dystrophy

Limb-girdle muscular dystrophy type 2D/R3 (LGMD2D/R3) is a progressive muscular dystrophy that manifests with muscle weakness, respiratory abnormalities, and in rare cases cardiomyopathy. LGMD2D/R3 is caused by mutations in the SGCA gene resulting in loss of protein and concomitant loss of some or a...

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Detalles Bibliográficos
Autores principales: Griffin, Danielle A., Pozsgai, Eric R., Heller, Kristin N., Potter, Rachael A., Peterson, Ellyn L., Rodino-Klapac, Louise R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2021
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8066346/
https://www.ncbi.nlm.nih.gov/pubmed/33349138
http://dx.doi.org/10.1089/hum.2019.199

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8066346/
https://www.ncbi.nlm.nih.gov/pubmed/33349138
http://dx.doi.org/10.1089/hum.2019.199

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