An alternative splicing modulator decreases mutant HTT and improves the molecular fingerprint in Huntington’s disease patient neurons

Huntington’s disease (HD) is a neurodegenerative disorder caused by poly-Q expansion in the Huntingtin (HTT) protein. Here, we delineate elevated mutant HTT (mHTT) levels in patient-derived cells including fibroblasts and iPSC derived cortical neurons using mesoscale discovery (MSD) HTT assays. HD p...

Descripción completa

Detalles Bibliográficos
Autores principales: Krach, Florian, Stemick, Judith, Boerstler, Tom, Weiss, Alexander, Lingos, Ioannis, Reischl, Stephanie, Meixner, Holger, Ploetz, Sonja, Farrell, Michaela, Hehr, Ute, Kohl, Zacharias, Winner, Beate, Winkler, Juergen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9649613/
https://www.ncbi.nlm.nih.gov/pubmed/36357392
http://dx.doi.org/10.1038/s41467-022-34419-x

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9649613/
https://www.ncbi.nlm.nih.gov/pubmed/36357392
http://dx.doi.org/10.1038/s41467-022-34419-x

Ejemplares similares