Cargando…

Splicing mutations in the CFTR gene as therapeutic targets

The marketing approval, about ten years ago, of the first disease modulator for patients with cystic fibrosis harboring specific CFTR genotypes (~5% of all patients) brought new hope for their treatment. To date, several therapeutic strategies have been approved and the number of CFTR mutations targ...

Descripción completa

Detalles Bibliográficos
Autores principales: Deletang, Karine, Taulan-Cadars, Magali
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9385490/
https://www.ncbi.nlm.nih.gov/pubmed/35650428
http://dx.doi.org/10.1038/s41434-022-00347-0