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Gene Therapy Applications of Non-Human Lentiviral Vectors

Recent commercialization of lentiviral vector (LV)-based cell therapies and successful reports of clinical studies have demonstrated the untapped potential of LVs to treat diseases and benefit patients. LVs hold notable and inherent advantages over other gene transfer agents based on their ability t...

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Detalles Bibliográficos
Autor principal: Munis, Altar M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7599719/
https://www.ncbi.nlm.nih.gov/pubmed/33003635
http://dx.doi.org/10.3390/v12101106
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author Munis, Altar M.
author_facet Munis, Altar M.
author_sort Munis, Altar M.
collection PubMed
description Recent commercialization of lentiviral vector (LV)-based cell therapies and successful reports of clinical studies have demonstrated the untapped potential of LVs to treat diseases and benefit patients. LVs hold notable and inherent advantages over other gene transfer agents based on their ability to transduce non-dividing cells, permanently transform target cell genome, and allow stable, long-term transgene expression. LV systems based on non-human lentiviruses are attractive alternatives to conventional HIV-1-based LVs due to their lack of pathogenicity in humans. This article reviews non-human lentiviruses and highlights their unique characteristics regarding virology and molecular biology. The LV systems developed based on these lentiviruses, as well as their successes and shortcomings, are also discussed. As the field of gene therapy is advancing rapidly, the use of LVs uncovers further challenges and possibilities. Advances in virology and an improved understanding of lentiviral biology will aid in the creation of recombinant viral vector variants suitable for translational applications from a variety of lentiviruses.
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spelling pubmed-75997192020-11-01 Gene Therapy Applications of Non-Human Lentiviral Vectors Munis, Altar M. Viruses Review Recent commercialization of lentiviral vector (LV)-based cell therapies and successful reports of clinical studies have demonstrated the untapped potential of LVs to treat diseases and benefit patients. LVs hold notable and inherent advantages over other gene transfer agents based on their ability to transduce non-dividing cells, permanently transform target cell genome, and allow stable, long-term transgene expression. LV systems based on non-human lentiviruses are attractive alternatives to conventional HIV-1-based LVs due to their lack of pathogenicity in humans. This article reviews non-human lentiviruses and highlights their unique characteristics regarding virology and molecular biology. The LV systems developed based on these lentiviruses, as well as their successes and shortcomings, are also discussed. As the field of gene therapy is advancing rapidly, the use of LVs uncovers further challenges and possibilities. Advances in virology and an improved understanding of lentiviral biology will aid in the creation of recombinant viral vector variants suitable for translational applications from a variety of lentiviruses. MDPI 2020-09-29 /pmc/articles/PMC7599719/ /pubmed/33003635 http://dx.doi.org/10.3390/v12101106 Text en © 2020 by the author. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Munis, Altar M.
Gene Therapy Applications of Non-Human Lentiviral Vectors
title Gene Therapy Applications of Non-Human Lentiviral Vectors
title_full Gene Therapy Applications of Non-Human Lentiviral Vectors
title_fullStr Gene Therapy Applications of Non-Human Lentiviral Vectors
title_full_unstemmed Gene Therapy Applications of Non-Human Lentiviral Vectors
title_short Gene Therapy Applications of Non-Human Lentiviral Vectors
title_sort gene therapy applications of non-human lentiviral vectors
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7599719/
https://www.ncbi.nlm.nih.gov/pubmed/33003635
http://dx.doi.org/10.3390/v12101106
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