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Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing

CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/Cas9 system as a ribonucleoprotein (RNP) comp...

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Autores principales: Zhang, Song, Shen, Jiangtao, Li, Dali, Cheng, Yiyun
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Ivyspring International Publisher 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7738854/
https://www.ncbi.nlm.nih.gov/pubmed/33391496
http://dx.doi.org/10.7150/thno.47007
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author Zhang, Song
Shen, Jiangtao
Li, Dali
Cheng, Yiyun
author_facet Zhang, Song
Shen, Jiangtao
Li, Dali
Cheng, Yiyun
author_sort Zhang, Song
collection PubMed
description CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/Cas9 system as a ribonucleoprotein (RNP) complex consisting of Cas9 protein and single guide RNA (sgRNA) has emerged as a powerful and widespread method for genome editing due to its advantages of transient genome editing and reduced off-target effects. In this review, we summarized the current Cas9 RNP delivery systems including physical approaches and synthetic carriers. The mechanisms and beneficial roles of these strategies in intracellular Cas9 RNP delivery were reviewed. Examples in the development of stimuli-responsive and targeted carriers for RNP delivery are highlighted. Finally, the challenges of current Cas9 RNP delivery systems and perspectives in rational design of next generation materials for this promising field will be discussed.
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spelling pubmed-77388542021-01-01 Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing Zhang, Song Shen, Jiangtao Li, Dali Cheng, Yiyun Theranostics Review CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years, however, the translation of this biotechnology into therapy has been hindered by efficient delivery of CRISPR/Cas9 materials into target cells. Direct delivery of CRISPR/Cas9 system as a ribonucleoprotein (RNP) complex consisting of Cas9 protein and single guide RNA (sgRNA) has emerged as a powerful and widespread method for genome editing due to its advantages of transient genome editing and reduced off-target effects. In this review, we summarized the current Cas9 RNP delivery systems including physical approaches and synthetic carriers. The mechanisms and beneficial roles of these strategies in intracellular Cas9 RNP delivery were reviewed. Examples in the development of stimuli-responsive and targeted carriers for RNP delivery are highlighted. Finally, the challenges of current Cas9 RNP delivery systems and perspectives in rational design of next generation materials for this promising field will be discussed. Ivyspring International Publisher 2021-01-01 /pmc/articles/PMC7738854/ /pubmed/33391496 http://dx.doi.org/10.7150/thno.47007 Text en © The author(s) This is an open access article distributed under the terms of the Creative Commons Attribution License (https://creativecommons.org/licenses/by/4.0/). See http://ivyspring.com/terms for full terms and conditions.
spellingShingle Review
Zhang, Song
Shen, Jiangtao
Li, Dali
Cheng, Yiyun
Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title_full Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title_fullStr Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title_full_unstemmed Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title_short Strategies in the delivery of Cas9 ribonucleoprotein for CRISPR/Cas9 genome editing
title_sort strategies in the delivery of cas9 ribonucleoprotein for crispr/cas9 genome editing
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7738854/
https://www.ncbi.nlm.nih.gov/pubmed/33391496
http://dx.doi.org/10.7150/thno.47007
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