Journal article
Advances in gene therapy for cystic fibrosis lung disease
Human molecular genetics, Vol.28(R1), pp.R88-R94
10/01/2019
DOI: 10.1093/hmg/ddz139
PMCID: PMC6796993
PMID: 31332440
Abstract
Cystic fibrosis (CF) is a multiorgan recessive genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Gene therapy efforts have focused on treating the lung, since it manifests the most significant life-threatening disease. Over two decades have past since the first CF lung gene therapy trials and significant advances in the therapeutic implementation of pharmacologic CFTR modulators have renewed the field's focus on developing gene therapies for the 10% of CF patients these modulators cannot help. This review summarizes recent progress made in developing vectors for airway transduction and CF animal models required for understanding the relevant cellular targets in the lung and testing the efficacy of gene therapy approaches. We also highlight future opportunities in emerging gene editing strategies that may offer advantages for treating diseases like CF where the gene target is highly regulated at the cellular level. The outcomes of CF lung gene therapy trials will likely inform productive paths toward gene therapy for other complex genetic disorders, while also advancing treatments for all CF patients.
Details
- Title: Subtitle
- Advances in gene therapy for cystic fibrosis lung disease
- Creators
- Ziying Yan - University of IowaPaul B. McCray - Roy J. and Lucille A. Carver College of MedicineJohn F. Engelhardt - University of Iowa
- Resource Type
- Journal article
- Publication Details
- Human molecular genetics, Vol.28(R1), pp.R88-R94
- DOI
- 10.1093/hmg/ddz139
- PMID
- 31332440
- PMCID
- PMC6796993
- NLM abbreviation
- Hum Mol Genet
- ISSN
- 0964-6906
- eISSN
- 1460-2083
- Publisher
- Oxford Univ Press
- Number of pages
- 7
- Grant note
- Cystic Fibrosis Foundation; Italian Cystic Fibrosis Research Foundation DK54759 / University of Iowa Center for Gene Therapy P30ES005605 / NATIONAL INSTITUTE OF ENVIRONMENTAL HEALTH SCIENCES; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Environmental Health Sciences (NIEHS) HL051670; HL91842; HL123482 / National Institutes of Health; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA P30DK054759 / NATIONAL INSTITUTE OF DIABETES AND DIGESTIVE AND KIDNEY DISEASES; United States Department of Health & Human Services; National Institutes of Health (NIH) - USA; NIH National Institute of Diabetes & Digestive & Kidney Diseases (NIDDK) Roy J. Carver Chair in Molecular Medicine Roy J. Carver Chair in Pulmonary Research
- Language
- English
- Date published
- 10/01/2019
- Academic Unit
- Roy J. Carver Department of Biomedical Engineering; Microbiology and Immunology; Pulmonary Medicine; Anatomy and Cell Biology; Stead Family Department of Pediatrics; Radiation Oncology; Internal Medicine
- Record Identifier
- 9984284335502771
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