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AAV-mediated gene therapy: Advancing cardiovascular disease treatment

Gene therapy has revolutionized the field of medicine, offering new hope for those with common and rare diseases. For nearly three decades, adeno-associated virus (AAV) has shown significant therapeutic benefits in multiple clinical trials, mainly due to its unique replication defects and non-pathog...

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Autores principales: Zhang, Huili, Zhan, Qi, Huang, Biao, Wang, Yigang, Wang, Xiaoyan
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Frontiers Media S.A. 2022
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9437345/
https://www.ncbi.nlm.nih.gov/pubmed/36061546
http://dx.doi.org/10.3389/fcvm.2022.952755
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author Zhang, Huili
Zhan, Qi
Huang, Biao
Wang, Yigang
Wang, Xiaoyan
author_facet Zhang, Huili
Zhan, Qi
Huang, Biao
Wang, Yigang
Wang, Xiaoyan
author_sort Zhang, Huili
collection PubMed
description Gene therapy has revolutionized the field of medicine, offering new hope for those with common and rare diseases. For nearly three decades, adeno-associated virus (AAV) has shown significant therapeutic benefits in multiple clinical trials, mainly due to its unique replication defects and non-pathogenicity in humans. In the field of cardiovascular disease (CVD), compared with non-viral vectors, lentiviruses, poxviruses, and adenovirus vectors, AAV possesses several advantages, including high security, low immunogenicity, sustainable and stable exogenous gene expression etc., which makes AAV one of the most promising candidates for the treatment of many genetic disorders and hereditary diseases. In this review, we evaluate the current information on the immune responses, transport pathways, and mechanisms of action associated with AAV-based CVD gene therapies and further explore potential optimization strategies to improve the efficiency of AAV transduction for the improved safety and efficiency of CVD treatment. In conclusion, AAV-mediated gene therapy has great potential for development in the cardiovascular system.
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spelling pubmed-94373452022-09-03 AAV-mediated gene therapy: Advancing cardiovascular disease treatment Zhang, Huili Zhan, Qi Huang, Biao Wang, Yigang Wang, Xiaoyan Front Cardiovasc Med Cardiovascular Medicine Gene therapy has revolutionized the field of medicine, offering new hope for those with common and rare diseases. For nearly three decades, adeno-associated virus (AAV) has shown significant therapeutic benefits in multiple clinical trials, mainly due to its unique replication defects and non-pathogenicity in humans. In the field of cardiovascular disease (CVD), compared with non-viral vectors, lentiviruses, poxviruses, and adenovirus vectors, AAV possesses several advantages, including high security, low immunogenicity, sustainable and stable exogenous gene expression etc., which makes AAV one of the most promising candidates for the treatment of many genetic disorders and hereditary diseases. In this review, we evaluate the current information on the immune responses, transport pathways, and mechanisms of action associated with AAV-based CVD gene therapies and further explore potential optimization strategies to improve the efficiency of AAV transduction for the improved safety and efficiency of CVD treatment. In conclusion, AAV-mediated gene therapy has great potential for development in the cardiovascular system. Frontiers Media S.A. 2022-08-19 /pmc/articles/PMC9437345/ /pubmed/36061546 http://dx.doi.org/10.3389/fcvm.2022.952755 Text en Copyright © 2022 Zhang, Zhan, Huang, Wang and Wang. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
spellingShingle Cardiovascular Medicine
Zhang, Huili
Zhan, Qi
Huang, Biao
Wang, Yigang
Wang, Xiaoyan
AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title_full AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title_fullStr AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title_full_unstemmed AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title_short AAV-mediated gene therapy: Advancing cardiovascular disease treatment
title_sort aav-mediated gene therapy: advancing cardiovascular disease treatment
topic Cardiovascular Medicine
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9437345/
https://www.ncbi.nlm.nih.gov/pubmed/36061546
http://dx.doi.org/10.3389/fcvm.2022.952755
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