AAV9-mediated Schwann cell-targeted gene therapy rescues a model of demyelinating neuropathy

Mutations in the GJB1 gene, encoding the gap junction (GJ) protein connexin32 (Cx32), cause X-linked Charcot-Marie-Tooth disease (CMT1X), an inherited demyelinating neuropathy. We developed a gene therapy approach for CMT1X using an AAV9 vector to deliver the GJB1/Cx32 gene under the myelin protein...

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Detalles Bibliográficos
Autores principales: Kagiava, Alexia, Karaiskos, Christos, Richter, Jan, Tryfonos, Christina, Jennings, Matthew J., Heslegrave, Amanda J., Sargiannidou, Irene, Stavrou, Marina, Zetterberg, Henrik, Reilly, Mary M., Christodoulou, Christina, Horvath, Rita, Kleopa, Kleopas A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8599011/
https://www.ncbi.nlm.nih.gov/pubmed/33692503
http://dx.doi.org/10.1038/s41434-021-00250-0

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8599011/
https://www.ncbi.nlm.nih.gov/pubmed/33692503
http://dx.doi.org/10.1038/s41434-021-00250-0

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