Study type: In vitro · Status: Evidence verified by machine 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: Not published. The text held for this field did not answer this heading, so nothing is shown here and nothing is substituted for it.
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.
Public plain-language summary
Some fields are not published, because the text held for them did not answer their heading. The published text is limited to the declared source and preserves reported uncertainty. It does not establish efficacy, safety, suitability, or evidence strength.
Research question
In this study, we explored the hypothesis that delivery of ABE using novel self-assembling receptor targeted lipid nanoparticles (RTN) and conversion of G542X to G542R could lead to a therapeutically functional treatment.
Study design
Not published. The text held for this field did not answer this heading, so nothing is shown here and nothing is substituted for it.
Participants or experimental system
We used an adenine base editor (ABE8e Cas9) and guide RNA (sgRNA)/enhanced green fluorescent protein (EGFP) plasmids encapsulated in receptor targeted nanoparticles (RTN), delivered to Bmi-1 transduced basal human CF airway epithelial cells harbouring the homozygous CFTR G542X variant, to convert the stop codon to G542R, a variant which is amenable to modulator therapy.
Study scale
Not published. The text held for this field did not answer this heading, so nothing is shown here and nothing is substituted for it.
Intervention or exposure
52% editing alone or 17% and 52% editing of CFTR G542X plus treatment with CFTR modulators (VX-445/VX-661/VX-770; ETI/Trikafta/Kaftrio) increased epithelial CFTR protein expression, CFTR protein band C abundance, CFTR172 inhibitable anion transport, and changes in airway surface liquid height and pH in response to vasoactive intestinal peptide (VIP) stimulation.
Comparator
Not published. The text held for this field did not answer this heading, so nothing is shown here and nothing is substituted for it.
Outcomes examined
We assessed editing efficiency of ABE delivered as plasmid DNA also encoding EGFP, by RTN to CFTR G542X homozygous airway epithelial cells.
Key findings
This results in nonsense-mediated decay and loss of functional CFTR protein which leads to defective anion transport and the development of CF disease pathology.
Limitations and uncertainty
Not published. The text held for this field did not answer this heading, so nothing is shown here and nothing is substituted for it.
Product relevance and evidence boundary
This record is a published study, held here with its citation and review status. It is not a statement that any catalog item is effective, safe, or suitable for any use, and nothing in it is a dose or a protocol.
Evidence verified by machine 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.