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Gene replacement therapy in two Golgi-retained CMT1X mutants before and after the onset of demyelinating neuropathy
X-linked Charcot-Marie-Tooth disease type 1 (CMT1X) is a demyelinating neuropathy resulting from loss-of-function mutations affecting the GJB1/connexin 32 (Cx32) gene. We previously showed functional and morphological improvement in Gjb1-null mice following AAV9-mediated delivery of human Cx32 drive...
| Autores principales: | , , , , , , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
| Publicado: |
American Society of Gene & Cell Therapy
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10460951/ https://www.ncbi.nlm.nih.gov/pubmed/37645436 http://dx.doi.org/10.1016/j.omtm.2023.07.011 |