Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy

Le Wang; Wenfu Zheng; Shaoqin Liu; Bing Li; Xingyu Jiang

doi:10.1002/cbic.201800629

Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy

Le Wang
, Wenfu Zheng^*
, Shaoqin Liu
, Bing Li
, Xingyu Jiang

^*Corresponding author for this work

School of Life Science and Technology, Harbin Institute of Technology
National Center for Nanoscience and Technology
Jinan University
Southern University of Science and Technology

Research output: Contribution to journal › Review article › peer-review

60 Scopus citations

Abstract

Precise editing of the genome of a living body is a goal pursued by scientists in many fields. In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas (CRISPR-associated) genome-editing systems have become a revolutionary toolbox for gene editing across various species. However, the low transfection efficiency of the CRISPR/Cas9 system to mammalian cells in vitro and in vivo is a big obstacle hindering wide and deep application. In this review, recently developed delivery strategies for various CRISPR/Cas9 formulations and their applications in treating gene-related diseases are briefly summarized. This review should inspire others to explore more efficient strategies for CRISPR system delivery and gene therapy.

Original language	English
Pages (from-to)	634-643
Number of pages	10
Journal	ChemBioChem
Volume	20
Issue number	5
DOIs	https://doi.org/10.1002/cbic.201800629
State	Published - 1 Mar 2019
Externally published	Yes

Keywords

carriers
delivery systems
gene technology
gene therapy
nanomaterials

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10.1002/cbic.201800629

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title = "Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy",

abstract = "Precise editing of the genome of a living body is a goal pursued by scientists in many fields. In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas (CRISPR-associated) genome-editing systems have become a revolutionary toolbox for gene editing across various species. However, the low transfection efficiency of the CRISPR/Cas9 system to mammalian cells in vitro and in vivo is a big obstacle hindering wide and deep application. In this review, recently developed delivery strategies for various CRISPR/Cas9 formulations and their applications in treating gene-related diseases are briefly summarized. This review should inspire others to explore more efficient strategies for CRISPR system delivery and gene therapy.",

keywords = "carriers, delivery systems, gene technology, gene therapy, nanomaterials",

author = "Le Wang and Wenfu Zheng and Shaoqin Liu and Bing Li and Xingyu Jiang",

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year = "2019",

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doi = "10.1002/cbic.201800629",

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AU - Wang, Le

AU - Zheng, Wenfu

AU - Liu, Shaoqin

AU - Li, Bing

AU - Jiang, Xingyu

PY - 2019/3/1

Y1 - 2019/3/1

N2 - Precise editing of the genome of a living body is a goal pursued by scientists in many fields. In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas (CRISPR-associated) genome-editing systems have become a revolutionary toolbox for gene editing across various species. However, the low transfection efficiency of the CRISPR/Cas9 system to mammalian cells in vitro and in vivo is a big obstacle hindering wide and deep application. In this review, recently developed delivery strategies for various CRISPR/Cas9 formulations and their applications in treating gene-related diseases are briefly summarized. This review should inspire others to explore more efficient strategies for CRISPR system delivery and gene therapy.

AB - Precise editing of the genome of a living body is a goal pursued by scientists in many fields. In recent years, CRISPR (clustered regularly interspaced short palindromic repeat)/Cas (CRISPR-associated) genome-editing systems have become a revolutionary toolbox for gene editing across various species. However, the low transfection efficiency of the CRISPR/Cas9 system to mammalian cells in vitro and in vivo is a big obstacle hindering wide and deep application. In this review, recently developed delivery strategies for various CRISPR/Cas9 formulations and their applications in treating gene-related diseases are briefly summarized. This review should inspire others to explore more efficient strategies for CRISPR system delivery and gene therapy.

KW - carriers

KW - delivery systems

KW - gene technology

KW - gene therapy

KW - nanomaterials

UR - https://www.scopus.com/pages/publications/85058220567

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DO - 10.1002/cbic.201800629

M3 - 文献综述

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VL - 20

SP - 634

EP - 643

JO - ChemBioChem

JF - ChemBioChem

IS - 5

ER -

Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy

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Keywords

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