ArticleMolecular therapy : the journal of the American Society of Gene Therapy2025
Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.
Article in Molecular therapy : the journal of the American Society of Gene Therapy, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 2 papers.
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2 citing papers in PubMed.
- Combining Gene Therapy with Current Modulator Treatments for Cystic Fibrosis: A Promising Area of Research.Pharmaceutics · 2026Review
- Ano1 Antisense Therapy Improves Enamel Properties in Molars but not Amelogenesis in a Murine Model of Cystic Fibrosis.Calcified tissue international · 2026Article
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11 authors.
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Abstract
Patients with cystic fibrosis (CF) who are non-responsive to treatments due to specific mutations need alternative CF transmembrane conductance regulator (CFTR)-independent therapies. This study aims to assess the impact of TMEM16a expression by a specific oligonucleotide (TMEM16a ASO) on dysregulated parameters in CF, which will help prepare for preclinical studies. In this study, we analyzed the effects of TMEM16a oligonucleotide within a CF context by evaluating the impact, optimal administration route, toxicity, and specificity in primary cells and various mouse models. The oligonucleotide enhances TMEM16a activity, increases Cl
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