ArticleMolecular therapy. Nucleic acids2025
Antisense oligonucleotide targeting the E3 ligase RFFL potentiates CFTR modulator efficacy in CF primary bronchial epithelial cells.
Article in Molecular therapy. Nucleic acids, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 1 paper.
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1 citing paper in PubMed.
- RFFL antisense oligonucleotides: A novel stabilization strategy to broaden the reach of CFTR modulator therapy.Molecular therapy. Nucleic acids · 2026Article
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10 authors.
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Abstract
Cystic fibrosis (CF) is most commonly caused by the ΔF508 mutation in the CFTR gene, leading to misfolding and degradation of the CFTR protein. Although CFTR modulators such as elexacaftor/tezacaftor/ivacaftor (ETI) provide clinical benefit, their efficacy is limited, particularly in patients with rare or poorly responsive CFTR mutations. RFFL, an E3 ubiquitin ligase, plays a central role in peripheral quality control of CFTR, reducing its plasma membrane (PM) expression and attenuating the effects of modulators. Here, we developed antisense oligonucleotides (ASOs) containing artificial nucleic acids to selectively suppress RFFL expression. An optimized
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