{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,26]],"date-time":"2026-09-26T13:57:27Z","timestamp":1790431047556,"version":"4.1.0"},"reference-count":153,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2019,3,14]],"date-time":"2019-03-14T00:00:00Z","timestamp":1552521600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100011352","name":"Center for Gene Therapy, University of Iowa","doi-asserted-by":"publisher","award":["NIH P30 DK 054759"],"award-info":[{"award-number":["NIH P30 DK 054759"]}],"id":[{"id":"10.13039\/100011352","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["NIH P01 HL-51670, NIH P01 HL-091842, NIH R01 HL-133089, NIH R01 HL-105821, NIH R44 HL-139218, NIH T32GM007337"],"award-info":[{"award-number":["NIH P01 HL-51670, NIH P01 HL-091842, NIH R01 HL-133089, NIH R01 HL-105821, NIH R44 HL-139218, NIH T32GM007337"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000897","name":"Cystic Fibrosis Foundation","doi-asserted-by":"publisher","award":["511141"],"award-info":[{"award-number":["511141"]}],"id":[{"id":"10.13039\/100000897","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Despite the continued development of cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs for the treatment of cystic fibrosis (CF), the need for mutation agnostic treatments remains. In a sub-group of CF individuals with mutations that may not respond to modulators, such as those with nonsense mutations, CFTR gene transfer to airway epithelia offers the potential for an effective treatment. Lentiviral vectors are well-suited for this purpose because they transduce nondividing cells, and provide long-term transgene expression. Studies in primary cultures of human CF airway epithelia and CF animal models demonstrate the long-term correction of CF phenotypes and low immunogenicity using lentiviral vectors. Further development of CF gene therapy requires the investigation of optimal CFTR expression in the airways. Lentiviral vectors with improved safety features have minimized insertional mutagenesis safety concerns raised in early clinical trials for severe combined immunodeficiency using \u03b3-retroviral vectors. Recent clinical trials using improved lentiviral vectors support the feasibility and safety of lentiviral gene therapy for monogenetic diseases. While work remains to be done before CF gene therapy reaches the bedside, recent advances in lentiviral vector development reviewed here are encouraging and suggest it could be tested in clinical studies in the near future.<\/jats:p>","DOI":"10.3390\/genes10030218","type":"journal-article","created":{"date-parts":[[2019,3,15]],"date-time":"2019-03-15T04:12:09Z","timestamp":1552623129000},"page":"218","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":57,"title":["Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease"],"prefix":"10.3390","volume":"10","author":[{"given":"Laura I.","family":"Marquez Loza","sequence":"first","affiliation":[{"name":"Stead Family Department of Pediatrics, The University of Iowa, Iowa City, IA 52242, USA"},{"name":"Pappajohn Biomedical Institute and the Center for Gene Therapy, The University of Iowa, Iowa City, IA 52242, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eric C.","family":"Yuen","sequence":"additional","affiliation":[{"name":"Talee Bio, 3001 Market Street, Suite 140, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"suffix":"Jr.","given":"Paul B.","family":"McCray","sequence":"additional","affiliation":[{"name":"Stead Family Department of Pediatrics, The University of Iowa, Iowa City, IA 52242, USA"},{"name":"Pappajohn Biomedical Institute and the Center for Gene Therapy, The University of Iowa, Iowa City, IA 52242, USA"},{"name":"Talee Bio, 3001 Market Street, Suite 140, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2019,3,14]]},"reference":[{"key":"ref_1","first-page":"8","article-title":"Ivacaftor: A novel gene-based therapeutic approach for cystic fibrosis","volume":"18","author":"Condren","year":"2013","journal-title":"J. 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