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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Veröffentlicht in:Nat Neurosci
Hauptverfasser: Van Alstyne, Meaghan, Tattoli, Ivan, Delestree, Nicolas, Recinos, Yocelyn, Workman, Eileen, Shihabuddin, Lamya S., Zhang, Chaolin, Mentis, George Z., Pellizzoni, Livio
Format: Artigo
Sprache:Inglês
Veröffentlicht: 2021
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Article
Online Zugang: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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