Gene Therapy for Spinal Muscular Atrophy: An Emerging Treatment Option for a Devastating Disease

BACKGROUND: Spinal muscular atrophy (SMA) is an autosomal recessive neurodegenerative disease that, in most cases, involves homozygous deletion of the SMN1 gene. This causes a deficiency in survival motor neuron (SMN) protein, which plays a critical role in motor neuron development. SMA has a range...

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Detalles Bibliográficos
Autores principales: Rao, Vamshi K., Kapp, Daniel, Schroth, Mary
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Academy of Managed Care Pharmacy 2018
Materias:
Supplement
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10408414/
https://www.ncbi.nlm.nih.gov/pubmed/30582825
http://dx.doi.org/10.18553/jmcp.2018.24.12-a.s3

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