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Npj Drug Discovery|July 1, 2026
A novel drug series optimized to address cystic fibrosis and other CFTR deficiency diseases of human airwaysAndras Rab, Jeong S Hong, Candela Manfredi, et al.
Biochemistry|December 12, 2025
Barriers to the Pharmacologic Rescue of W1282X CFTRCandela Manfredi, Andras Rab, Disha Joshi, et al.
Scientific Reports|February 27, 2026
Large variations in total and allele-specific transcript expression in a disease mutation-independent mannerMoritz Freyberg, Merete Bewig, Giovana Bavia Bampi, et al.
ACS Medicinal Chemistry Letters|October 18, 2023
A Novel 7<i>H</i>-[1,2,4]Triazolo[3,4-<i>b</i>]thiadiazine-based Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Directed toward Treatment of Cystic FibrosisAndras Rab, Xun Yang, William F Tracy, et al.
Biorxiv : the Preprint Server for Biology|January 23, 2024
Development of robust antiviral assays using relevant apical-out human airway organoidsJi-Hoon Lee, Julia C LeCher, Eric Parigoris, et al.
Plos One|February 14, 2025
Evaluating elexacaftor/tezacaftor/ivacaftor (ETI; Trikafta™) for treatment of patients with non-cystic fibrosis bronchiectasis (NCFBE): A clinical study protocolColin E Swenson, William R Hunt, Candela Manfredi, et al.
The Lancet. Respiratory Medicine|August 29, 2024
Evaluation of elexacaftor-tezacaftor-ivacaftor treatment in individuals with cystic fibrosis and CFTR<sup>N1303K</sup> in the USA: a prospective, multicentre, open-label, single-arm trialGeorge M Solomon, Rachel W Linnemann, Rachel Rich, et al.
Journal of Virology|July 24, 2020
Type I and Type III Interferons Restrict SARS-CoV-2 Infection of Human Airway Epithelial CulturesAbigail Vanderheiden, Philipp Ralfs, Tatiana Chirkova, et al.
Nature|May 31, 2023
Engineered tRNAs suppress nonsense mutations in cells and in vivoSuki Albers, Elizabeth C Allen, Nikhil Bharti, et al.
FASEB Journal : Official Publication of the Federation of American Societies for Experimental Biology|October 6, 2023
Mechanisms by which the cystic fibrosis transmembrane conductance regulator may influence SARS-CoV-2 infection and COVID-19 disease severityPhilip R Tedbury, Candela Manfredi, Frauke Degenhardt, et al.
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