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Gene therapy rescues disease phenotype in a spinal muscular atrophy with respiratory distress type 1 (SMARD1) mouse model

Spinal muscular atrophy with respiratory distress type 1 (SMARD1) is an autosomal recessive motor neuron disease affecting children. It is caused by mutations in the IGHMBP2 gene (11q13) and presently has no cure. Recently, adeno-associated virus serotype 9 (AAV9)–mediated gene therapy has been show...

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Detalles Bibliográficos
Autores principales:	Nizzardo, Monica, Simone, Chiara, Rizzo, Federica, Salani, Sabrina, Dametti, Sara, Rinchetti, Paola, Del Bo, Roberto, Foust, Kevin, Kaspar, Brian K., Bresolin, Nereo, Comi, Giacomo P., Corti, Stefania
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Association for the Advancement of Science 2015
Materias:	Research Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4643829/ https://www.ncbi.nlm.nih.gov/pubmed/26601156 http://dx.doi.org/10.1126/sciadv.1500078

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