Systematic review and meta-analysis determining the benefits of in vivo genetic therapy in spinal muscular atrophy rodent models

Spinal muscular atrophy (SMA) is a severe childhood neuromuscular disease for which two genetic therapies, Nusinersen (Spinraza, an antisense oligonucleotide), and AVXS-101 (Zolgensma, an adeno-associated viral vector of serotype 9 AAV9), have recently been approved. We investigated the pre-clinical...

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Detalles Bibliográficos
Autores principales: Chilcott, Ellie M., Muiruri, Evalyne W., Hirst, Theodore C., Yáñez-Muñoz, Rafael J.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9482879/
https://www.ncbi.nlm.nih.gov/pubmed/34611322
http://dx.doi.org/10.1038/s41434-021-00292-4

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9482879/
https://www.ncbi.nlm.nih.gov/pubmed/34611322
http://dx.doi.org/10.1038/s41434-021-00292-4

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