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Motor unit recovery following Smn restoration in mouse models of spinal muscular atrophy

Spinal muscular atrophy (SMA) is a childhood motor neuron disease caused by anomalies in the SMN1 gene. Although therapeutics have been approved for the treatment of SMA, there is a therapeutic time window, after which efficacy is reduced. Hallmarks of motor unit pathology in SMA include loss of mot...

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Detalles Bibliográficos
Autores principales: Comley, Laura H, Kline, Rachel A, Thomson, Alison K, Woschitz, Victoria, Landeros, Eric Villalón, Osman, Erkan Y, Lorson, Christian L, Murray, Lyndsay M
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9476628/
https://www.ncbi.nlm.nih.gov/pubmed/35551393
http://dx.doi.org/10.1093/hmg/ddac097