Intraperitoneal delivery of a novel drug‐like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder that causes progressive muscle weakness and is the leading genetic cause of infant mortality worldwide. SMA is caused by the loss of survival motor neuron 1 (SMN1). In humans, a nearly identical copy gene is present,...

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Detalles Bibliográficos
Autores principales: Osman, E. Y., Rietz, A., Kline, R. A., Cherry, J. J., Hodgetts, K. J., Lorson, C. L., Androphy, E. J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6367425/
https://www.ncbi.nlm.nih.gov/pubmed/30733501
http://dx.doi.org/10.1038/s41598-018-38208-9

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