CFTR

(642) CFTR Rescue in Intestinal Organoids with FT Triple Combination in Comparison to Trikafta

(643) Delivery of gp64-pseudotyped lentivirus carrying codon-optimized CFTR provides improved functional restoration in human CF airway epithelial cultures

(644) Identification of organoid responders to CFTR modulators in the HIT-CF Europe project – Underlying the need for new treatment strategies for people with ultra-rare mutations

(645) A Novel eRF1 Degrader Induces Translational Readthrough of CFTR Nonsense Mutations to Therapeutically Relevant Levels in Combination with Aminoglycosides

(647) Potentiator Icenticaftor Is Superior to Ivacaftor in Rescuing F508del CFTR and Does Not Destabilize Corrected Mature Protein

(648) Distinct Proteostasis and Structural States Drive VX-809 Susceptibility of CFTR Mutants

(650) Ribosomal Silencing Confers CFTR Nonsense Suppression and is Synergistic with Highly Effective Modulators

(651) Hyperglycemia Contributes to Oxidative Stress and Impairs Potency of Highly Effective Modulator Therapies.

(652) Electrophysiological approaches to study CFTR function from human induced pluripotent stem cells (iPSC)-derived airway Basal Cells.

(653) Targeting CFTR ubiquitination to augment therapeutic strategies

(654) Mechanism of Pulmonary Vascular Dysfunction in Cystic Fibrosis.

(655) Respiratory cell-type affinity and absolute CFTR expression in the primate airway upon nebulization of KB407

(657) Combinatory treatment of LAU-7b with triple therapy results in improved lung physiology and restored fatty acid and lipid levels

(658) Characterization of CFTR-expressing Epithelial Cell Lineages in Mouse Airways by Fate Mapping

(659) Inflammation Augments Trikafta-Induced CFTR Rescue in Primary Airway Epithelia Carrying F508del and Rare CFTR Mutations

(660) Triple potentiator-mediated activation of CFTR mutants with gating defects is independent of channel phosphorylation and NBD dimerization

(661) The PDE4 Inhibitor Apremilast Enhances Benefits of CFTR Modulators in vitro and in vivo

(664) Effectiveness and safety of elexacaftor-tezacaftor-ivacaftor therapy: an Italian center experience

(665) Single-cell RNA sequencing reveals pulmonary ionocytes subtypes in proximal ferret airway epithelium

(666) Ivacaftor Boosts CFTR-Mediated Nasal Potential Difference in CFTR Knockout Mice Treated with hCFTR-mRNA Lipid Nanoparticle

(667) EMC is Required for CFTR Biogenesis and Activation in vivo

(668) CRISPR based cellular models with endogenous expression of HiBiT tagged wild type and mutant CFTR enables high-throughput biology studies and screening for new transporter modulators

(669) Conformational Dynamics of CFTR Revealed by Single-Molecule FRET

(670) TGF-β1 diminishes efficacy of highly effective CFTR modulators, an effect reversed by losartan

(671) Adenylate cyclase 6 interactome regulates mucociliary clearance

(673) Trikafta^TM corrects function of H1085R-, N1303K- and R334W-CFTR and improves clinical status of patients.

(674) Loss of a functional CFTR leads to a calpain-dependent degradation of ezrin in activated CF macrophages

(675) Personalized medicine approaches directed at rare CFTR trafficking mutations contribute to elucidation of correction mechanisms

(677) Impact of Highly effective modulator therapy (HEMT) on the CF Microbiome

(678) Role of Adenylate Cyclase 6 autophagy complex in the rescue and function of F508del CFTR protein

(679) Transduction of Ferret Airway, Liver and Pancreas Following a Single Dose of AAV1

(680) A strategy to edit disease-causing, segmental duplications in CFTR

(681) Assessing CFTR function in respiratory epithelial cell cultures derived from nasal brushings: a new tool to assess individual treatment responses?

(683) Characteristics of CFTR Modulator Response in Nasal Cell Models Derived from Patients with Rare CFTR Variants

(685) CryoEM reconstruction of the conformational ensemble adopted by hCFTR-G551D provides insight into the folding pathway and the mechanism-of-action of the dual corrector/potentiator Elexacaftor (VX445)

(689) Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR

(691) Synthetic aminoglycoside ELX-02 induces readthrough of CFTR-G550X producing super-functional protein that can be further enhanced by CFTR correctors

(692) The importance of CFTR mRNA testing to uncover other variants in CF genotype that affect CFTR expression