Muscle overexpression of Klf15 via an AAV8-Spc5-12 construct does not provide benefits in spinal muscular atrophy mice

Spinal muscular atrophy (SMA) is a neuromuscular disease caused by loss of the survival motor neuron (SMN) gene. While there are currently two approved gene-based therapies for SMA, availability, high cost, and differences in patient response indicate that alternative treatment options are needed. O...

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Detalles Bibliográficos
Autores principales: Ahlskog, Nina, Hayler, Daniel, Krueger, Anja, Kubinski, Sabrina, Claus, Peter, Hammond, Suzan M., Wood, Matthew J. A., Yáñez-Muñoz, Rafael J., Bowerman, Melissa
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7674152/
https://www.ncbi.nlm.nih.gov/pubmed/32313099
http://dx.doi.org/10.1038/s41434-020-0146-8

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7674152/
https://www.ncbi.nlm.nih.gov/pubmed/32313099
http://dx.doi.org/10.1038/s41434-020-0146-8

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