Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.

doi:10.1016/j.jcf.2021.06.003

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Article

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Sept 2021

Oren Yifat S, Irony-Tur Sinai Michal, Golec Anita, Barchad-Avitzur Ofra, Mutyam Venkateshwar, Li Yao, Hong Jeong, Ozeri-Galai Efrat, Hatton Aurélie, Leibson Chen, Carmel Liran, Reiter Joel, Sorscher Eric J, Wilton Steve D, Kerem Eitan, Rowe Steven M, Sermet-Gaudelus Isabelle, Kerem Batsheva

Abstract excerpt

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 in the CFTR gene. METHODS: We have screened, in FRT cells expressing the 3849+10 kb C-to-T splicing mutation, ~30...

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Identifiers

PMID: 34226157
DOI: 10.1016/j.jcf.2021.06.003
PMCID: PMC8464507

Genes

CFTR

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