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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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| Publicado no: | Nat Neurosci |
|---|---|
| Main Authors: | , , , , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2021
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| Assuntos: | |
| Acesso em linha: | 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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