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Recombinant Adeno-Associated Virus Serotype 9 Gene Therapy in Spinal Muscular Atrophy

Spinal muscular atrophy (SMA) is an autosomal recessive neuromuscular disease caused by deletion or mutation of the SMN1 gene. It is characterized by a progressive loss of motor neurons resulting in muscle weakness. The disease affects 1 in 11,000 live births and before the era of treatment SMA was...

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Detalles Bibliográficos
Autores principales:	Kotulska, Katarzyna, Fattal-Valevski, Aviva, Haberlova, Jana
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Frontiers Media S.A. 2021
Materias:	Neurology
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8548432/ https://www.ncbi.nlm.nih.gov/pubmed/34721262 http://dx.doi.org/10.3389/fneur.2021.726468

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