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Article

Intestinal organoids as a platform for functional evaluation of ASO-mediated splicing modulation in cystic fibrosis.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jul 2026

Stanleigh Noemie, Irony-Tur Sinai Michal, Oren Yifat, Ozeri-Galai Efrat, Hart Gili, Grunewald Myriam, Birimberg-Schwartz Liron, Beekman Jeffrey M, Kerem Eitan, Wilschanski Michael, Kerem Batsheva

Abstract excerpt

BACKGROUND: CFTR modulating therapies have advanced significantly but remain ineffective for people with CF (pwCF) with mutations preventing CFTR protein production. PwCF carrying non-canonical splicing mutations show modest benefits. SPL84, an antisense oligonucleotide (ASO) developed by Splisense, targets the non-canonical CFTR splicing mutation 3849+10 kb C→T (3849) and is currently in Phase2 trials. SPL84-23,...

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Share to Cystic Fibrosis
Share to Cystic Fibrosis Transmembrane Conductance Regulator

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to CFTR gene

Share to Mutation

Share to Oligonucleotides, Antisense

Share to Organoids

Share to RNA Splicing

Source

Identifiers

PMID: 41506934
DOI: 10.1016/j.jcf.2026.01.001

Genes

CFTR

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 4 Sept 2026.

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