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Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects

Gene therapy has been experiencing a breakthrough in recent years, targeting various specific cell groups in numerous therapeutic areas. However, most recent clinical studies maintain the use of traditional viral vector systems, which are challenging to manufacture cost-effectively at a commercial s...

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Detalles Bibliográficos
Autores principales: Zu, Hui, Gao, Danchen
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer International Publishing 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8171234/
https://www.ncbi.nlm.nih.gov/pubmed/34076797
http://dx.doi.org/10.1208/s12248-021-00608-7
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author Zu, Hui
Gao, Danchen
author_facet Zu, Hui
Gao, Danchen
author_sort Zu, Hui
collection PubMed
description Gene therapy has been experiencing a breakthrough in recent years, targeting various specific cell groups in numerous therapeutic areas. However, most recent clinical studies maintain the use of traditional viral vector systems, which are challenging to manufacture cost-effectively at a commercial scale. Non-viral vectors have been a fast-paced research topic in gene delivery, such as polymers, lipids, inorganic particles, and combinations of different types. Although non-viral vectors are low in their cytotoxicity, immunogenicity, and mutagenesis, attracting more and more researchers to explore the promising delivery system, they do not carry ideal characteristics and have faced critical challenges, including gene transfer efficiency, specificity, gene expression duration, and safety. This review covers the recent advancement in non-viral vectors research and formulation aspects, the challenges, and future perspectives.
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spelling pubmed-81712342021-06-02 Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects Zu, Hui Gao, Danchen AAPS J Review Article Gene therapy has been experiencing a breakthrough in recent years, targeting various specific cell groups in numerous therapeutic areas. However, most recent clinical studies maintain the use of traditional viral vector systems, which are challenging to manufacture cost-effectively at a commercial scale. Non-viral vectors have been a fast-paced research topic in gene delivery, such as polymers, lipids, inorganic particles, and combinations of different types. Although non-viral vectors are low in their cytotoxicity, immunogenicity, and mutagenesis, attracting more and more researchers to explore the promising delivery system, they do not carry ideal characteristics and have faced critical challenges, including gene transfer efficiency, specificity, gene expression duration, and safety. This review covers the recent advancement in non-viral vectors research and formulation aspects, the challenges, and future perspectives. Springer International Publishing 2021-06-02 /pmc/articles/PMC8171234/ /pubmed/34076797 http://dx.doi.org/10.1208/s12248-021-00608-7 Text en © American Association of Pharmaceutical Scientists 2021 This article is made available via the PMC Open Access Subset for unrestricted research re-use and secondary analysis in any form or by any means with acknowledgement of the original source. These permissions are granted for the duration of the World Health Organization (WHO) declaration of COVID-19 as a global pandemic.
spellingShingle Review Article
Zu, Hui
Gao, Danchen
Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title_full Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title_fullStr Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title_full_unstemmed Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title_short Non-viral Vectors in Gene Therapy: Recent Development, Challenges, and Prospects
title_sort non-viral vectors in gene therapy: recent development, challenges, and prospects
topic Review Article
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8171234/
https://www.ncbi.nlm.nih.gov/pubmed/34076797
http://dx.doi.org/10.1208/s12248-021-00608-7
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