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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Springer International Publishing
2021
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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. |
format | Online Article Text |
id | pubmed-8171234 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Springer International Publishing |
record_format | MEDLINE/PubMed |
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 |
work_keys_str_mv | AT zuhui nonviralvectorsingenetherapyrecentdevelopmentchallengesandprospects AT gaodanchen nonviralvectorsingenetherapyrecentdevelopmentchallengesandprospects |