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Restoring SMN Expression: An Overview of the Therapeutic Developments for the Treatment of Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disorder and one of the most common genetic causes of infant death. It is characterized by progressive weakness of the muscles, loss of ambulation, and death from respiratory complications. SMA is caused by the homozygous dele...

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Detalles Bibliográficos
Autores principales:	Aslesh, Tejal, Yokota, Toshifumi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2022
Materias:	Review
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8834523/ https://www.ncbi.nlm.nih.gov/pubmed/35159227 http://dx.doi.org/10.3390/cells11030417

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