AAV1.NT-3 gene therapy for X-linked Charcot–Marie–Tooth neuropathy type 1

X-linked Charcot-Marie-Tooth neuropathy (CMTX) is caused by mutations in the gene encoding Gap Junction Protein Beta-1 (GJB1)/Connexin32 (Cx32) in Schwann cells. Neurotrophin-3 (NT-3) is an important autocrine factor supporting Schwann cell survival and differentiation and stimulating axon regenerat...

Descripción completa

Detalles Bibliográficos
Autores principales: Ozes, Burcak, Myers, Morgan, Moss, Kyle, Mckinney, Jennifer, Ridgley, Alicia, Chen, Lei, Bai, Shasha, Abrams, Charles K., Freidin, Mona M., Mendell, Jerry R., Sahenk, Zarife
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/PMC9013664/
https://www.ncbi.nlm.nih.gov/pubmed/33542455
http://dx.doi.org/10.1038/s41434-021-00231-3

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9013664/
https://www.ncbi.nlm.nih.gov/pubmed/33542455
http://dx.doi.org/10.1038/s41434-021-00231-3

Ejemplares similares