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Adeno-associated virus gene therapy to the rescue for Charcot-Marie-Tooth disease type 4J
The genetic peripheral neuropathy known as Charcot-Marie-Tooth disease type 4J (CMT4J) is caused by recessive mutations in the FIG4 gene. The transformational success of adeno-associated virus (AAV) gene therapy for spinal muscular atrophy has generated substantial interest in using this approach to...
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| Publicat a: | J Clin Invest |
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| Autor principal: | |
| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
American Society for Clinical Investigation
2021
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| Matèries: | |
| Accés en línia: | https://ncbi.nlm.nih.gov/pmc/articles/PMC8159695/ https://ncbi.nlm.nih.gov/pubmed/34060476 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI149492 |
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