Journal article
Considerations for early life genetic therapies in cystic fibrosis
American journal of physiology. Lung cellular and molecular physiology
04/29/2026
DOI: 10.1152/ajplung.00022.2026
PMID: 42053522
Abstract
Cystic fibrosis (CF) is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR) anion channel. Since the initial characterization of CF in the early 20
century, advances in clinical management have reduced disease burden and increased longevity. These advances are amplified by the recent development of highly effective CFTR modulator therapies (HEMT) that provided remarkable clinical results in people with CF (pwCF). However, some pwCF do not benefit from HEMT due to several limiting factors. Genetic therapies have increasingly emerged as prospective, complementary treatments to HEMTs for those unable to benefit from HEMT. Genetic therapies have yet to be approved for clinical use in pwCF, but multiple clinical trials are in progress. In anticipation of future approval of one or more of these candidates, it is essential to identify and discuss how these genetic therapies might be used in early life, when many CF lesions originate, especially for those with severe mutations. In this multidisciplinary review, we discuss several pertinent factors such as CFTR localization and function, CF disease origins, emerging developments in genetic therapies, ethical considerations and other perspectives needed to guide future genetic therapies in early life.
Details
- Title: Subtitle
- Considerations for early life genetic therapies in cystic fibrosis
- Creators
- Ashley L Cooney - University of IowaIan M Thornell - University of IowaAlejandro A Pezzulo - University of IowaAnthony J Fischer - University of IowaDaniel P Cook - University of IowaMahmoud H Abou Alaiwa - Internal Medicine, Roy J and Lucille A. Carver College of Medicine, University of Iowa, Iowa City, Iowa USAKatie M Larson Ode - University of IowaAliye Uc - University of IowaJoseph Zabner - University of IowaAmy L Ryan - University of IowaDavid A Stoltz - University of IowaPatrick L Sinn - University of IowaPaul B McCray Jr - University of Iowa, Pulmonary MedicineDavid K Meyerholz - University of Iowa
- Resource Type
- Journal article
- Publication Details
- American journal of physiology. Lung cellular and molecular physiology
- DOI
- 10.1152/ajplung.00022.2026
- PMID
- 42053522
- ISSN
- 1522-1504
- eISSN
- 1522-1504
- Publisher
- American Physiological Society
- Grant note
- HL133089 / HHS | NIH | National Heart, Lung, and Blood Institute (NHLBI) K08AI181763 / HHS | NIH | National Heart, Lung, and Blood Institute (NHLBI) P01 HL152960 / NHLBI NIH HHS THORNE24G0 / Cystic Fibrosis Foundation (CFF) HL171035 / HHS | NIH | National Heart, Lung, and Blood Institute (NHLBI) COOK20L0 / Cystic Fibrosis Foundation (CFF) FISCHE25G0 / Cystic Fibrosis Foundation (CFF) COOK24A0-KB / Cystic Fibrosis Foundation (CFF) COOK25R3 / Cystic Fibrosis Foundation (CFF) FISCHE24Y0 / Cystic Fibrosis Foundation (CFF)
- Language
- English
- Electronic publication date
- 04/29/2026
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Molecular Physiology and Biophysics; Pulmonary, Critical Care, and Occupational Medicine; Endocrinology and Diabetes; Microbiology and Immunology; Pulmonary Medicine; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Pathology; Iowa Neuroscience Institute; Gastroenterology, Hepatology, Pancreatology, and Nutrition; Internal Medicine
- Record Identifier
- 9985157615402771
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