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Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing

Genome-editing technology has emerged as a potential tool for treating incurable diseases for which few therapeutic modalities are available. In particular, discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system together with the design of single-guide RNAs (s...

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Autores principales: Kim, Dongyoon, Le, Quoc-Viet, Wu, Yina, Park, Jinwon, Oh, Yu-Kyoung
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2020
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7766488/
https://www.ncbi.nlm.nih.gov/pubmed/33353099
http://dx.doi.org/10.3390/pharmaceutics12121233
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author Kim, Dongyoon
Le, Quoc-Viet
Wu, Yina
Park, Jinwon
Oh, Yu-Kyoung
author_facet Kim, Dongyoon
Le, Quoc-Viet
Wu, Yina
Park, Jinwon
Oh, Yu-Kyoung
author_sort Kim, Dongyoon
collection PubMed
description Genome-editing technology has emerged as a potential tool for treating incurable diseases for which few therapeutic modalities are available. In particular, discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system together with the design of single-guide RNAs (sgRNAs) has sparked medical applications of genome editing. Despite the great promise of the CRISPR/Cas system, its clinical application is limited, in large part, by the lack of adequate delivery technology. To overcome this limitation, researchers have investigated various systems, including viral and nonviral vectors, for delivery of CRISPR/Cas and sgRNA into cells. Among nonviral delivery systems that have been studied are nanovesicles based on lipids, polymers, peptides, and extracellular vesicles. These nanovesicles have been designed to increase the delivery of CRISPR/Cas and sgRNA through endosome escape or using various stimuli such as light, pH, and environmental features. This review covers the latest research trends in nonviral, nanovesicle-based delivery systems that are being applied to genome-editing technology and suggests directions for future progress.
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spelling pubmed-77664882020-12-28 Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing Kim, Dongyoon Le, Quoc-Viet Wu, Yina Park, Jinwon Oh, Yu-Kyoung Pharmaceutics Review Genome-editing technology has emerged as a potential tool for treating incurable diseases for which few therapeutic modalities are available. In particular, discovery of the clustered regularly interspaced short palindromic repeats (CRISPR)/Cas system together with the design of single-guide RNAs (sgRNAs) has sparked medical applications of genome editing. Despite the great promise of the CRISPR/Cas system, its clinical application is limited, in large part, by the lack of adequate delivery technology. To overcome this limitation, researchers have investigated various systems, including viral and nonviral vectors, for delivery of CRISPR/Cas and sgRNA into cells. Among nonviral delivery systems that have been studied are nanovesicles based on lipids, polymers, peptides, and extracellular vesicles. These nanovesicles have been designed to increase the delivery of CRISPR/Cas and sgRNA through endosome escape or using various stimuli such as light, pH, and environmental features. This review covers the latest research trends in nonviral, nanovesicle-based delivery systems that are being applied to genome-editing technology and suggests directions for future progress. MDPI 2020-12-18 /pmc/articles/PMC7766488/ /pubmed/33353099 http://dx.doi.org/10.3390/pharmaceutics12121233 Text en © 2020 by the authors. Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (http://creativecommons.org/licenses/by/4.0/).
spellingShingle Review
Kim, Dongyoon
Le, Quoc-Viet
Wu, Yina
Park, Jinwon
Oh, Yu-Kyoung
Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title_full Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title_fullStr Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title_full_unstemmed Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title_short Nanovesicle-Mediated Delivery Systems for CRISPR/Cas Genome Editing
title_sort nanovesicle-mediated delivery systems for crispr/cas genome editing
topic Review
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7766488/
https://www.ncbi.nlm.nih.gov/pubmed/33353099
http://dx.doi.org/10.3390/pharmaceutics12121233
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