AAV9-Mediated Expression of SMN Restricted to Neurons Does Not Rescue the Spinal Muscular Atrophy Phenotype in Mice

Spinal muscular atrophy (SMA) is a neuromuscular disease mainly caused by mutations or deletions in the survival of motor neuron 1 (SMN1) gene and characterized by the degeneration of motor neurons and progressive muscle weakness. A viable therapeutic approach for SMA patients is a gene replacement...

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Detalles Bibliográficos
Autores principales: Besse, Aurore, Astord, Stephanie, Marais, Thibaut, Roda, Marianne, Giroux, Benoit, Lejeune, François-Xavier, Relaix, Frederic, Smeriglio, Piera, Barkats, Martine, Biferi, Maria Grazia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2020
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7403319/
https://www.ncbi.nlm.nih.gov/pubmed/32470325
http://dx.doi.org/10.1016/j.ymthe.2020.05.011

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