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Lessons learned from lung and liver in-vivo gene therapy: implications for the future
Introduction: Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness. Areas covered: This a...
Autores principales: | , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Taylor & Francis
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6134476/ https://www.ncbi.nlm.nih.gov/pubmed/30067117 http://dx.doi.org/10.1080/14712598.2018.1506761 |
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author | van Haasteren, Joost Hyde, Stephen C. Gill, Deborah R. |
author_facet | van Haasteren, Joost Hyde, Stephen C. Gill, Deborah R. |
author_sort | van Haasteren, Joost |
collection | PubMed |
description | Introduction: Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness. Areas covered: This article provides a review of in-vivo approaches for gene therapy in the lung and liver, exploiting non-viral and viral vectors with varying serotypes and pseudotypes to target-specific cells. Antibody responses inhibiting viral vectors continue to constrain effective repeat administration. Lessons learned from ex-vivo gene therapy and genome editing are also discussed. Expert opinion: The fields of lung and liver in-vivo gene therapy are thriving and a comparison highlights obstacles and opportunities for both. Overcoming immunological issues associated with repeated administration of viral vectors remains a key challenge. The addition of targeted small molecules in combination with viral vectors may offer one solution. A substantial bottleneck to the widespread adoption of in-vivo gene therapy is how to ensure sufficient capacity for clinical-grade vector production. In the future, the exploitation of gene editing approaches for in-vivo disease treatment may facilitate the resurgence of non-viral gene transfer approaches, which tend to be eclipsed by more efficient viral vectors. |
format | Online Article Text |
id | pubmed-6134476 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2018 |
publisher | Taylor & Francis |
record_format | MEDLINE/PubMed |
spelling | pubmed-61344762018-09-20 Lessons learned from lung and liver in-vivo gene therapy: implications for the future van Haasteren, Joost Hyde, Stephen C. Gill, Deborah R. Expert Opin Biol Ther Review Introduction: Ex-vivo gene therapy has had significant clinical impact over the last couple of years and in-vivo gene therapy products are being approved for clinical use. Gene therapy and gene editing approaches have huge potential to treat genetic disease and chronic illness. Areas covered: This article provides a review of in-vivo approaches for gene therapy in the lung and liver, exploiting non-viral and viral vectors with varying serotypes and pseudotypes to target-specific cells. Antibody responses inhibiting viral vectors continue to constrain effective repeat administration. Lessons learned from ex-vivo gene therapy and genome editing are also discussed. Expert opinion: The fields of lung and liver in-vivo gene therapy are thriving and a comparison highlights obstacles and opportunities for both. Overcoming immunological issues associated with repeated administration of viral vectors remains a key challenge. The addition of targeted small molecules in combination with viral vectors may offer one solution. A substantial bottleneck to the widespread adoption of in-vivo gene therapy is how to ensure sufficient capacity for clinical-grade vector production. In the future, the exploitation of gene editing approaches for in-vivo disease treatment may facilitate the resurgence of non-viral gene transfer approaches, which tend to be eclipsed by more efficient viral vectors. Taylor & Francis 2018-08-10 /pmc/articles/PMC6134476/ /pubmed/30067117 http://dx.doi.org/10.1080/14712598.2018.1506761 Text en © 2018 Informa UK Limited, trading as Taylor & Francis Group http://creativecommons.org/licenses/by/4.0/ This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/4.0/), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. |
spellingShingle | Review van Haasteren, Joost Hyde, Stephen C. Gill, Deborah R. Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title | Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title_full | Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title_fullStr | Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title_full_unstemmed | Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title_short | Lessons learned from lung and liver in-vivo gene therapy: implications for the future |
title_sort | lessons learned from lung and liver in-vivo gene therapy: implications for the future |
topic | Review |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6134476/ https://www.ncbi.nlm.nih.gov/pubmed/30067117 http://dx.doi.org/10.1080/14712598.2018.1506761 |
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