Postsymptomatic restoration of SMN rescues the disease phenotype in a mouse model of severe spinal muscular atrophy

Spinal muscular atrophy (SMA) is a common neuromuscular disorder in humans. In fact, it is the most frequently inherited cause of infant mortality, being the result of mutations in the survival of motor neuron 1 (SMN1) gene that reduce levels of SMN protein. Restoring levels of SMN protein in indivi...

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Detalhes bibliográficos
Main Authors: Lutz, Cathleen M., Kariya, Shingo, Patruni, Sunita, Osborne, Melissa A., Liu, Don, Henderson, Christopher E., Li, Darrick K., Pellizzoni, Livio, Rojas, José, Valenzuela, David M., Murphy, Andrew J., Winberg, Margaret L., Monani, Umrao R.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2011
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3148744/
https://ncbi.nlm.nih.gov/pubmed/21785219
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI57291
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