Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

Variant-selective stereopure oligonucleotides protect against pathologies associated with C9orf72-repeat expansion in preclinical models

C9orf72 expansion mutations are the most common genetic cause of ALS and FTD, which have limited therapies. The authors generate stereopure oligonucleotides that selectively deplete expansion-containing transcripts and protect against expansion-associated pathologies in preclinical models.

Guardado en:
Detalles Bibliográficos
Autores principales: Yuanjing Liu, Jean-Cosme Dodart, Helene Tran, Shaunna Berkovitch, Maurine Braun, Michael Byrne, Ann F. Durbin, Xiao Shelley Hu, Naoki Iwamoto, Hyun Gyung Jang, Pachamuthu Kandasamy, Fangjun Liu, Kenneth Longo, Jörg Ruschel, Juili Shelke, Hailin Yang, Yuan Yin, Amy Donner, Zhong Zhong, Chandra Vargeese, Robert H. Brown
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
Materias:
Science
Q
Acceso en línea:https://doaj.org/article/c67a2cf526554819ac5be15eaecb07de
Etiquetas: Agregar Etiqueta
Sin Etiquetas, Sea el primero en etiquetar este registro!

Ejemplares similares

Ejemplares similares