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...

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Detalles Bibliográficos
Autores principales: Kagiava, Alexia, Karaiskos, Christos, Lapathitis, George, Heslegrave, Amanda, Sargiannidou, Irene, Zetterberg, Henrik, Bosch, Assumpció, Kleopa, Kleopas A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
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

Internet

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

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