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Genes 2019, 10(3), 218; https://doi.org/10.3390/genes10030218

Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease

1
Stead Family Department of Pediatrics, The University of Iowa, Iowa City, IA 52242, USA
2
Pappajohn Biomedical Institute and the Center for Gene Therapy, The University of Iowa, Iowa City, IA 52242, USA
3
Talee Bio, 3001 Market Street, Suite 140, Philadelphia, PA 19104, USA
*
Author to whom correspondence should be addressed.
Received: 20 February 2019 / Revised: 8 March 2019 / Accepted: 11 March 2019 / Published: 14 March 2019
(This article belongs to the Special Issue Cystic Fibrosis: Therapy and Genetics)
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PDF [327 KB, uploaded 23 March 2019]

Abstract

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 γ-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. View Full-Text
Keywords: Cystic fibrosis; CFTR; gene therapy; safety; lentivirus; lentiviral vectors Cystic fibrosis; CFTR; gene therapy; safety; lentivirus; lentiviral vectors
This is an open access article distributed under the Creative Commons Attribution License which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited (CC BY 4.0).
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Marquez Loza, L.I.; Yuen, E.C.; McCray, P.B., Jr. Lentiviral Vectors for the Treatment and Prevention of Cystic Fibrosis Lung Disease. Genes 2019, 10, 218.

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