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Gene therapy in neuromuscular disorders
Monogenic neuromuscular disorders are potentially treatable through gene therapy. Using viral vectors, a therapeutic transgene aims to restore normal levels of a protein not produced by the defective gene, or to silence a gene whose expression leads to toxic effects. Spinal Muscular Atrophy (SMA) is...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Academia Brasileira de Neurologia - ABNEURO
2022
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9491441/ https://www.ncbi.nlm.nih.gov/pubmed/35976325 http://dx.doi.org/10.1590/0004-282X-ANP-2022-S135 |