PATH TO A CURE

(590) Airway epithelial stem cell phenotype and function are regulated by culture condition

(591) Modulation of the DNP Interactome Enhances CFTR Gene Transfer in a CF Mouse Model

(593) A murine reporter system to assess delivery and duration of genome edited or transplanted cells

(594) Correction of a CFTR canonical splice site variant, 3120+1G >A, by adenine base editing

(597) AAV capsid modification to improve tropism for airway epithelial progenitor cells

(598) Alternate start site M265 allows 5’ nonsense variants to escape NMD so that readthrough and modulators can restore CFTR function

(599) iPSC-derived airway basal cells that exhibit increased competence for multipotent differentiation

(600) A multimodal iPSC platform for cystic fibrosis drug testing

(601) Effects of different combinations of CFTR targeted drugs on CFTR dependent sweating in Cystic Fibrosis (CF) patients.

(602) Optimization of CFTR^G551D Ferret Model for Early Synchronized Lung Microbial Colonization

(603) Base editing rescues G542X CFTR mutation

(604) SpliSense’s ASO SPL84-23-1 Properly Distributes and is Retained in CF-like Mice Lungs

(605) Beneficial Effects of SGLT1/2 dual inhibitor Phloridzin on Human iPSC-derived Lung Organoids of Cystic Fibrosis Class I Mutations

(606) Focused Clinical Trials of Modulator Response for Rare Cystic Fibrosis Genotypes

(608) Anti-CD20 permits secondary lung gene transfer: implications for gene editing approaches in CF

(610) Evaluating the mechanism of amino acid insertion upon readthrough of CFTR nonsense mutations

(612) Optimized nanoparticle-mediated base editing of CF-causing variants

(613) Understanding synergy in nonsense suppression therapy for CFTR nonsense mutations

(615) Antisense Oligonucleotide-Based Drug Development for Cystic Fibrosis Patients Carrying the 2789+5 G-to-A Splicing Mutation

(616) CF Gene Therapy: Moving forward

(617) Peptide-Mediated Delivery of Adenine Base Editors to Rhesus Monkey Airway Epithelia

(618) SPL84-23-1 Antisense Oligonucleotide Modulating the 3849+10kb C-T Splicing Defect, Has an Additive Effect with TRIKAFTA in Primary HBE Cells from 3849 Patients

(619) Antisense Oligonucleotide Splicing Modulation as a Novel Cystic Fibrosis Therapeutic Approach for the W1282X Nonsense Mutation

(620) Antisense Oligonucleotide Splicing Modulation as a Novel Cystic Fibrosis Therapeutic Approach for the N1303K Nonsense Mutation

(622) DEVELOPMENT OF IN VITRO TRANSCRIBED mRNA THERAPEUTICS FOR CYSTIC FIBROSIS

(623) Preliminary assessment of nasal and colon-derived primary cells theratyping for a personalized medicine approach in CF patients carrying ultra-rare genotypes.

(624) Elexacaftor, tezacaftor, and ivacaftor for the treatment of cystic fibrosis: experience in Argentina from October 2021 to March 2022.

(625) Manufacturing and Device Development for SPL84-23-1, an Inhaled Antisense Oligonucleotide, Supporting a First-in-Human Clinical Study in CF Patients Carrying the 3849+10kb C-to-T mutation

(627) Temporal-spatial CRISPR base editing to identify target cells for genetic-based therapies

(629) Assessing the efficacy of airway gene therapy in neonatal rats

(630) Cellular Tropism of Adeno-associated Viral Vector 4 (AAV4) in Human Large and Small Airway Epithelia

(632) Evaluation of Gene Insertion Strategies for Restoration of CFTR Expression in Airway Epithelium

(633) Extracellular vesicles as delivery vectors to enhance killing of bacteria and viruses by airway-recruited human neutrophils

(636) Increased nonsense suppressor tRNA readthrough potency through sequence optimization

(638) Splice-switching Antisense Oligonucleotides for the Treatment of Class I CFTR Mutations

(640) The Cystic Fibrosis Mouse Model Resource Center