Systemic delivery of AAVrh74.tMCK.hCAPN3 rescues the phenotype in a mouse model for LGMD2A/R1

Limb girdle muscular dystrophy (LGMD) 2A/R1, caused by mutations in the CAPN3 gene and CAPN3 loss of function, is known to play a role in disease pathogenicity. In this study, AAVrh74.tMCK.CAPN3 was delivered systemically to two different age groups of CAPN3 knockout (KO) mice; each group included t...

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Detalles Bibliográficos
Autores principales: Sahenk, Zarife, Ozes, Burcak, Murrey, Darren, Myers, Morgan, Moss, Kyle, Yalvac, Mehmet E., Ridgley, Alicia, Chen, Lei, Mendell, Jerry R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8413669/
https://www.ncbi.nlm.nih.gov/pubmed/34514031
http://dx.doi.org/10.1016/j.omtm.2021.06.010

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8413669/
https://www.ncbi.nlm.nih.gov/pubmed/34514031
http://dx.doi.org/10.1016/j.omtm.2021.06.010

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