Study type: In vitro · Status: Verified against declared source
Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cells.
Cellular and molecular life sciences : CMLS · 2025
Study scale: Cystic fibrosis (CF) disease is a recessively inherited genetic disorder of the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes an anion channel.
Abstract only: Open source record
Product or molecular entity relationships
- VIP: Exact entity relationship. Legacy citation custody associates this source with the catalog record; no product-relevance conclusion is implied.
Plain-language verified summary
Question
The introduction of gene editing technologies, particularly CRISPR–Cas9, has heralded a new era in the potential treatment of genetic diseases like CF.
Methods
The online version contains supplementary material available at 10.1007/s00018-025-05587-y.
Scale or participants
Cystic fibrosis (CF) disease is a recessively inherited genetic disorder of the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes an anion channel.
Key findings
The cystic fibrosis (CF) causing variant G542X harbours a premature translation stop signal in the cystic fibrosis transmembrane conductance regulator (CFTR) mRNA.
Limitations and uncertainty
Cystic fibrosis (CF) disease is a recessively inherited genetic disorder of the cystic fibrosis transmembrane conductance regulator (CFTR) gene which encodes an anion channel.
Verified against declared source. Verification is limited to the declared source and review scope. It does not mean independent replication or establish efficacy, safety, or suitability.