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Amelioration of Muscle and Nerve Pathology in LAMA2 Muscular Dystrophy by AAV9-Mini-Agrin

LAMA2-related muscular dystrophy (LAMA2 MD) is the most common and fatal form of early-onset congenital muscular dystrophies. Due to the large size of the laminin α2 cDNA and heterotrimeric structure of the protein, it is challenging to develop a gene-replacement therapy. Our group has developed a n...

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Detalles Bibliográficos
Autores principales: Qiao, Chunping, Dai, Yi, Nikolova, Viktoriya D., Jin, Quan, Li, Jianbin, Xiao, Bin, Li, Juan, Moy, Sheryl S., Xiao, Xiao
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5948311/
https://www.ncbi.nlm.nih.gov/pubmed/29766020
http://dx.doi.org/10.1016/j.omtm.2018.01.005