A translatable RNAi-driven gene therapy silences PMP22/Pmp22 genes and improves neuropathy in CMT1A mice

Charcot-Marie-Tooth disease type 1A (CMT1A), the most common inherited demyelinating peripheral neuropathy, is caused by PMP22 gene duplication. Overexpression of WT PMP22 in Schwann cells destabilizes the myelin sheath, leading to demyelination and ultimately to secondary axonal loss and disability...

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Detalles Bibliográficos
Autores principales: Stavrou, Marina, Kagiava, Alexia, Choudury, Sarah G., Jennings, Matthew J., Wallace, Lindsay M., Fowler, Allison M., Heslegrave, Amanda, Richter, Jan, Tryfonos, Christina, Christodoulou, Christina, Zetterberg, Henrik, Horvath, Rita, Harper, Scott Q., Kleopa, Kleopas A.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Clinical Investigation 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9246392/
https://www.ncbi.nlm.nih.gov/pubmed/35579942
http://dx.doi.org/10.1172/JCI159814

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9246392/
https://www.ncbi.nlm.nih.gov/pubmed/35579942
http://dx.doi.org/10.1172/JCI159814

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