ArticleScience translational medicine2019
In utero and postnatal VX-770 administration rescues multiorgan disease in a ferret model of cystic fibrosis.
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Article in Science translational medicine, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 94 papers.
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94citing papers in PubMed
14.3field-weighted citation impact, top 1% of its field
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Who cites it
94 citing papers in PubMed, 179 citations in OpenAlex.
- Structural and functional impact of cystic fibrosis on endocrine pancreatic innervation.Clinical science (London, England : 1979) · 2026Article
- Male reproductive phenotype in cystic fibrosis: comparison of existing animal models.Lab animal · 2026Review
- Long-term effectiveness and safety of cystic fibrosis transmembrane conductance regulator modulators and their impact on the lives of people with cystic fibrosis.American journal of respiratory and critical care medicine · 2026Article
- Impact of Genetics on Endocrine Manifestations of Cystic Fibrosis.Endocrine practice : official journal of the American College of Endocrinology and the American Association of Clinical Endocrinologists · 2026Review
- Unravelling the developmental origins of cystic fibrosis-related diabetes.Diabetologia · 2026Review
- A therapeutic revolution: CFTR modulators in cystic fibrosis and their impacts on pregnant women and the fetus.Journal of perinatology : official journal of the California Perinatal Association · 2026Review
- Maternal and fetal outcomes associated with CFTR modulator use during pregnancy: a scoping review.European respiratory review : an official journal of the European Respiratory Society · 2026Article
- Considerations for early life genetic therapies in cystic fibrosis.American journal of physiology. Lung cellular and molecular physiology · 2026Review
- The evolving understanding of cystic fibrosis-related diabetes in the highly effective modulator therapy era: a scoping review.Diabetologia · 2026Article
- Review
- The role of ER-associated degradation and ER-phagy in health and disease.Signal transduction and targeted therapy · 2026Review
- Ferrets and genetically modified ferrets as model organisms in biomedical research: a review.Frontiers in genome editing · 2026Review
- Restoring chloride efflux in cystic fibrosis with TMEM16a antisense oligonucleotides.Molecular therapy : the journal of the American Society of Gene Therapy · 2025Article
- A ferret model demonstrates sustained fibrosis, restrictive physiology, and airway proximalization.NPJ Regenerative medicine · 2025Article
- Ferret model of bleomycin-induced lung injury shares features of human idiopathic pulmonary fibrosis.NPJ Regenerative medicine · 2025Article
- Profiling bile acids in the stools of humans and animal models of cystic fibrosis.Microbiology spectrum · 2025Article
- Ionocyte CFTR Coordinates Chloride Absorption and Secretion to Balance Airway Fluid.American journal of respiratory and critical care medicine · 2025Article
- Diagnosis and Emerging Biomarkers of Cystic Fibrosis-Related Kidney Disease (CFKD).Journal of clinical medicine · 2025Review
- Novel Cystic Fibrosis Ferret Model Enables Visualization of CFTR Expression Cells and Genetic CFTR Reactivation.Human gene therapy · 2025Article
- Recombinant Adeno-Associated Virus Vector Mediated Gene Editing in Proliferating and Polarized Cultures of Human Airway Epithelial Cells.Human gene therapy · 2025Article
34 more citing papers are in PubMed but not listed here.
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Authors and funding
24 authors at 3 institutions in 2 countries.
Xingshen SunDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.ORCID 0000-0003-3561-8428
Yaling YiDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.ORCID 0000-0001-5385-063X
Ziying YanDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.ORCID 0000-0001-8210-5567
Bradley H RosenDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.ORCID 0000-0001-5216-2632
Bo LiangDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Michael C WinterDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
T Idil Apak EvansDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Pavana G RottiDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Yu YangDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.ORCID 0000-0002-0043-0890
Jaimie S GrayDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Soo Yeun ParkDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Weihong ZhouDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Yulong ZhangDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Shashanna R MollDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
Lisa WoodyVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.ORCID 0000-0002-3169-8048
Dao M TranVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.
Licong JiangVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.ORCID 0000-0001-9193-5730
Annelotte M VonkPediatric Pulmonology and Regenerative Medicine Center, University Medical Center Utrecht, Utrecht, Netherlands.ORCID 0000-0003-1182-4518
Jeffrey M BeekmanPediatric Pulmonology and Regenerative Medicine Center, University Medical Center Utrecht, Utrecht, Netherlands.
Paul NegulescuVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.
Fred Van GoorVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.
Dennis F FiorinoVertex Pharmaceuticals Incorporated, San Diego, CA 92121, USA.ORCID 0000-0003-4324-1157
Katherine N Gibson-CorleyDepartment of Pathology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA.
John F EngelhardtDepartment of Anatomy & Cell Biology, Carver College of Medicine, University of Iowa, Iowa City, IA 52242, USA. john-engelhardt@uiowa.edu.ORCID 0000-0003-2389-9277
University of Iowa · USVertex Pharmaceuticals (United States) · USUniversity Medical Center Utrecht · NL
Funding
Pulmonary Toxicology Facility CoreP30ES005605 · NIEHS · UNIVERSITY OF IOWA · PI Brandi Janssen · 1990 to 2026
$40.5MVector CoreP01HL051670 · NHLBI · UNIVERSITY OF IOWA · PI MCCRAY, PAUL B · 1993 to 2019
$36.5MVector Core-Core 2P30DK054759 · NIDDK · UNIVERSITY OF IOWA · PI Alejandro Antonio Pezzulo · 1998 to 2026
$30.5MEarly Pathogenesis of Cystic Fibrosis Related DiabetesR24DK096518 · NIDDK · UNIVERSITY OF IOWA · PI ENGELHARDT, JOHN F, MORAN, ANTOINETTE M. · 2012 to 2018
$10.1MNational Ferret Resource and Research Center on Lung DiseaseR24HL123482 · NHLBI · UNIVERSITY OF IOWA · PI ENGELHARDT, JOHN F · 2014 to 2018
$3.8MMODELS OF SUBMUCOSAL GLAND DEVELOPMENT AND FUNCTIONR01DK047967 · NIDDK · UNIVERSITY OF PENNSYLVANIA · PI ENGELHARDT, JOHN F · 1993 to 2020
$3.8MThe University of Iowa Clinical and Translational Science AwardKL2TR002536 · NCATS · UNIVERSITY OF IOWA · PI BASSUK, ALEXANDER G · 2018 to 2022
$3.3MUltra-low Dose, High Speed, High Resolution, Multispectral, Multi-Source MDCT ScaS10OD018526 · OD · UNIVERSITY OF IOWA · PI HOFFMAN, ERIC ALFRED · 2015 to 2015
$2.0MSplanchno-Hormonal Mechanisms of Cystic Fibrosis Related DiabetesR01DK115791 · NIDDK · UNIVERSITY OF IOWA · PI ENGELHARDT, JOHN F, NORRIS, ANDREW W · 2018 to 2021
$1.9MNCATS NIH HHS KL2 TR002536NHLBI NIH HHS P01 HL051670NHLBI NIH HHS R24 HL123482NIDDK NIH HHS P30 DK054759NIDDK NIH HHS R01 DK047967NIDDK NIH HHS R01 DK115791NIDDK NIH HHS R24 DK096518NIEHS NIH HHS P30 ES005605NIH HHS S10 OD018526
Abstract
Cystic fibrosis (CF) is a multiorgan disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (
Indexed as
AminophenolsAnimalsAnimals, Genetically ModifiedAnimals, NewbornBlood GlucoseChloride Channel AgonistsCystic FibrosisCystic Fibrosis Transmembrane Conductance RegulatorDisease Models, AnimalDisease ProgressionFemaleFerretsGene Knock-In TechniquesGenitalia, MaleGestational AgeHumansAminophenolsBlood GlucoseCFTR protein, humanChloride Channel AgonistsCystic Fibrosis Transmembrane Conductance RegulatorivacaftorQuinolones
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.