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Biochemistry|December 12, 2025
Barriers to the Pharmacologic Rescue of W1282X CFTRCandela Manfredi, Andras Rab, Disha Joshi, et al.
ACS Medicinal Chemistry Letters|October 18, 2023
A Novel 7H-[1,2,4]Triazolo[3,4-b]thiadiazine-based Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Directed toward Treatment of Cystic FibrosisAndras Rab, Xun Yang, William F Tracy, et al.
Plos One|April 16, 2014
Chymase mediates injury and mitochondrial damage in cardiomyocytes during acute ischemia/reperfusion in the dogJunying Zheng, Chih-Chang Wei, Naoki Hase, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 14, 2020
Highly Efficient Gene Editing of Cystic Fibrosis Patient-Derived Airway Basal Cells Results in Functional CFTR CorrectionShingo Suzuki, Ana M Crane, Varada Anirudhan, et al.
The Journal of Clinical Investigation|October 29, 2019
Slowing ribosome velocity restores folding and function of mutant CFTRKathryn E Oliver, Robert Rauscher, Marjolein Mijnders, et al.
JCI Insight|July 27, 2018
Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulatorsSangwoo T Han, Andras Rab, Matthew J Pellicore, 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 CFTRN1303K in the USA: a prospective, multicentre, open-label, single-arm trialGeorge M Solomon, Rachel W Linnemann, Rachel Rich, et al.
Cell Stem Cell|October 25, 2020
Derivation of Airway Basal Stem Cells from Human Pluripotent Stem CellsFinn J Hawkins, Shingo Suzuki, Mary Lou Beermann, et al.
Nature Communications|July 17, 2021
A small molecule that induces translational readthrough of CFTR nonsense mutations by eRF1 depletionJyoti Sharma, Ming Du, Eric Wong, et al.
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