Cargando…
Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849 + 10 kb C-to-T splicing mutation
BACKGROUND: Antisense oligonucleotide (ASO)-based drugs for splicing modulation were recently approved for various genetic diseases with unmet need. Here we aimed to develop an ASO-based splicing modulation therapy for Cystic Fibrosis (CF) patients carrying the 3849 + 10 kb C-to-T splicing mutation...
Autores principales: | , , , , , , , , , , , , , , , , , |
---|---|
Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
2021
|
Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8464507/ https://www.ncbi.nlm.nih.gov/pubmed/34226157 http://dx.doi.org/10.1016/j.jcf.2021.06.003 |