新型基因治疗策略递送 CRISPR/Cas9

Delivery of CRISPR/Cas9 by Novel Strategies for Gene Therapy.

机构信息

School of Life Science and Technology, Harbin Institute of Technology, 2 Yikuang Road, Nangang District, Harbin, 150001, China.

Beijing Engineering Research Center for BioNanotechnology, CAS Key Lab for Biological Effects of Nanomaterials and Nanosafety, CAS Center of Excellence for Nanoscience, National Center for NanoScience and Technology, Beijing, 100190, China.

出版信息

Chembiochem. 2019 Mar 1;20(5):634-643. doi: 10.1002/cbic.201800629. Epub 2018 Dec 11.

DOI:10.1002/cbic.201800629

PMID:30393919

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.

摘要

精确编辑活体基因组是许多领域科学家追求的目标。近年来，CRISPR（成簇规律间隔短回文重复）/Cas（CRISPR 相关）基因组编辑系统已成为各种物种基因编辑的革命性工具盒。然而，CRISPR/Cas9 系统在体外和体内转染哺乳动物细胞的效率低，是阻碍其广泛和深入应用的一大障碍。本文简要总结了近年来开发的各种 CRISPR/Cas9 制剂的递药策略及其在治疗基因相关疾病中的应用。本文应能启发他人探索更有效的 CRISPR 系统递药和基因治疗策略。