In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely man...

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Detalles Bibliográficos
Autores principales: Behr, Matthew, Zhou, Jing, Xu, Bing, Zhang, Hongwei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2021
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8424283/
https://www.ncbi.nlm.nih.gov/pubmed/34522582
http://dx.doi.org/10.1016/j.apsb.2021.05.020
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author Behr, Matthew
Zhou, Jing
Xu, Bing
Zhang, Hongwei
author_facet Behr, Matthew
Zhou, Jing
Xu, Bing
Zhang, Hongwei
author_sort Behr, Matthew
collection PubMed
description Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely manipulating cellular DNA sequences, the low efficiency of in vivo delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of in vivo delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives.
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spelling pubmed-84242832021-09-13 In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges Behr, Matthew Zhou, Jing Xu, Bing Zhang, Hongwei Acta Pharm Sin B Review Within less than a decade since its inception, CRISPR-Cas9-based genome editing has been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly anticipated that this revolutionary technology will bring novel therapeutic modalities to many diseases by precisely manipulating cellular DNA sequences, the low efficiency of in vivo delivery must be enhanced before its therapeutic potential can be fully realized. Here we discuss the most recent progress of in vivo delivery of CRISPR-Cas9 systems, highlight innovative viral and non-viral delivery technologies, emphasize outstanding delivery challenges, and provide the most updated perspectives. Elsevier 2021-08 2021-05-26 /pmc/articles/PMC8424283/ /pubmed/34522582 http://dx.doi.org/10.1016/j.apsb.2021.05.020 Text en © 2021 Chinese Pharmaceutical Association and Institute of Materia Medica, Chinese Academy of Medical Sciences. Production and hosting by Elsevier B.V. https://creativecommons.org/licenses/by-nc-nd/4.0/This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Review
Behr, Matthew
Zhou, Jing
Xu, Bing
Zhang, Hongwei
In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title_full In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title_fullStr In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title_full_unstemmed In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title_short In vivo delivery of CRISPR-Cas9 therapeutics: Progress and challenges
title_sort in vivo delivery of crispr-cas9 therapeutics: progress and challenges
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8424283/
https://www.ncbi.nlm.nih.gov/pubmed/34522582
http://dx.doi.org/10.1016/j.apsb.2021.05.020
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