Journal
COMPUTATIONAL AND STRUCTURAL BIOTECHNOLOGY JOURNAL
Volume 18, Issue -, Pages 2401-2415Publisher
ELSEVIER
DOI: 10.1016/j.csbj.2020.08.031
Keywords
CRISPR; Cas9; Genome editing; Human disease models; Rabbit; Gene therapy; Off target effects
Funding
- National Key Research and Development Program of China Stem Cell and Translational Research [2017YFA0105101]
- Program for Changjiang Scholars and Innovative Research Team in University [IRT_16R32]
- Strategic Priority Research Program of the Chinese Academy of Sciences [XDA16030501, XDA16030503]
- Key Research & Development Program of Guangzhou Regenerative Medicine and Health Guangdong Laboratory [2018GZR110104004]
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Genome editing is the modification of genomic DNA at a specific target site in a wide variety of cell types and organisms, including insertion, deletion and replacement of DNA, resulting in inactivation of target genes, acquisition of novel genetic traits and correction of pathogenic gene mutations. Due to the advantages of simple design, low cost, high efficiency, good repeatability and short-cycle, CRISPR-Cas systems have become the most widely used genome editing technology in molecular biology laboratories all around the world. In this review, an overview of the CRISPR-Cas systems will be introduced, including the innovations, the applications in human disease research and gene therapy, as well as the challenges and opportunities that will be faced in the practical application of CRISPR-Cas systems. (C) 2020 The Author(s). Published by Elsevier B.V. on behalf of Research Network of Computational and Structural Biotechnology.
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