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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Bibliografiske detaljer
Udgivet i:Nat Neurosci
Main Authors: Van Alstyne, Meaghan, Tattoli, Ivan, Delestree, Nicolas, Recinos, Yocelyn, Workman, Eileen, Shihabuddin, Lamya S., Zhang, Chaolin, Mentis, George Z., Pellizzoni, Livio
Format: Artigo
Sprog:Inglês
Udgivet: 2021
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8254787/
https://ncbi.nlm.nih.gov/pubmed/33795885
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41593-021-00827-3
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