Article

K978C CFTR restores essential epithelial function with greater efficiency than wildtype CFTR when expressed in CF airway cells

2022-08-13

Abstract excerpt

Class Ia/b CFTR variants cause severe cystic fibrosis (CF) lung disease in ~10% of CF patients and are untreatable with small molecule pharmaceuticals. Genetic replacement strategies offer a potential cure for all patients but so far, have displayed limited efficiency in vivo . We hypothesised that increasing protein abundance and/or activity of introduced CFTRs would more effectively restore function to CF bron...

Topics

Open a Topic to create a Post that cites this publication.

Identifiers and source

Literature Corpus work: e3c9d8ab-bc1a-56a8-8ea5-32bc65743c2a
DOI: 10.1101/2022.08.10.503368

Open publication

Related research

Semantic proximity does not establish scientific evidence.

Click a neighbor to travelStep 1 · 12 closest

K978C CFTR restores essential epithelial function with greater efficiency than wildtype CFTR when expressed in CF airway cellsDOI 10.1101/2022.08.10.503368Read

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.Semantic proximity Read

Beyond Kaftrio : mechanistic insights to maximize N1303K-CFTR rescueSemantic proximity Read

<i>In Vitro</i> Modulator Responsiveness of 655 <i>CFTR</i> Variants Found in People With CFSemantic proximity Read

High-Throughput Screening for Modulators of CFTR Activity Based on Genetically Engineered Cystic Fibrosis Disease-Specific iPSCsSemantic proximity Read

Adenine base editing of CFTR using receptor targeted nanoparticles restores function to G542X cystic fibrosis airway epithelial cellsSemantic proximity Read

CFTR potentiators partially restore channel function to A561E-CFTR, a cystic fibrosis mutant with a similar mechanism of dysfunction as F508del-CFTR.Semantic proximity Read

Relationships among CFTR expression, HCO 3 − secretion, and host defense may inform gene- and cell-based cystic fibrosis therapiesSemantic proximity Read

Increased CFTR expression and function from an optimized lentiviral vector for cystic fibrosis gene therapySemantic proximity Read

Novel amino-carbonitrile-pyrazole identified in a small molecule screen activates wild-type and ΔF508 cystic fibrosis transmembrane conductance regulator in the absence of a cAMP agonist.Semantic proximity Read

Fluorescence assay for simultaneous quantification of CFTR ion-channel function and plasma membrane proximitySemantic proximity Read

Highly Efficient Repair of the ΔF508 Mutation in Airway Stem Cells of Cystic Fibrosis Patients with Functional Rescue of the Differentiated EpitheliaSemantic proximity Read

Porosome reconstitution therapy: A biologic rescue from cystic fibrosisSemantic proximity Read