Gain of toxic function by long-term AAV9-mediated SMN overexpression in the sensory-motor circuit

The neurodegenerative disease spinal muscular atrophy (SMA) is caused by deficiency in the survival motor neuron (SMN) protein. Currently approved SMA treatments aim to restore SMN, but the potential for SMN expression beyond physiological levels is a unique feature of AAV9-SMN gene therapy. Here, w...

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Detalles Bibliográficos
Autores principales: Van Alstyne, Meaghan, Tattoli, Ivan, Delestree, Nicolas, Recinos, Yocelyn, Workman, Eileen, Shihabuddin, Lamya S., Zhang, Chaolin, Mentis, George Z., Pellizzoni, Livio
Formato: Online Artículo Texto
Lenguaje:English
Publicado: 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8254787/
https://www.ncbi.nlm.nih.gov/pubmed/33795885
http://dx.doi.org/10.1038/s41593-021-00827-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8254787/
https://www.ncbi.nlm.nih.gov/pubmed/33795885
http://dx.doi.org/10.1038/s41593-021-00827-3

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