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...

Disgrifiad llawn

Wedi'i Gadw mewn:
Manylion Llyfryddiaeth
Prif Awduron: 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.
Fformat: Artigo
Iaith:Inglês
Cyhoeddwyd: American Society for Clinical Investigation 2011
Pynciau:
Research Article
Mynediad Ar-lein: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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