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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...

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Autores principales: van Haasteren, Joost, Hyde, Stephen C., Gill, Deborah R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Taylor & Francis 2018
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.
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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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