CRISPR editing in biological and biomedical investigation

文献类型： 外文期刊

第一作者： Huang, Jiaojiao

作者： Huang, Jiaojiao;Zhao, Jianguo;Wang, Yanfang;Zhao, Jianguo

作者机构：

关键词： base editing;CRISPR;Cas;gene therapy;human diseases models;transcriptional regulation

期刊名称：JOURNAL OF CELLULAR PHYSIOLOGY （ 影响因子：6.384； 五年影响因子：5.987 ）

ISSN： 0021-9541

年卷期： 2018 年 233 卷 5 期

页码：

收录情况： SCI

摘要： Recently, clustered regularly interspaced short palindromic repeats (CRISPR) based genomic editing technologies have armed researchers with powerful new tools to biological and biomedical investigations. To further improve and expand its functionality, natural, and engineered CRISPR associated nine proteins (Cas9s) have been investigated, various CRISPR delivery strategies have been tested and optimized, and multiple schemes have been developed to ensure precise mammalian genome editing. Benefiting from those in-depth understanding and further development of CRISPR, versatile CRISPR-based platforms for genome editing have been rapidly developed to advance investigations in biology and biomedicine. In biological research area, CRISPR has been widely adopted in both fundamental and applied research fields, such as accurate base editing, transcriptional regulation, and genome-wide screening. In biomedical research area, CRISPR has also shown its extensive applicability in the establishment of animal models for genetic disorders especially those large animals and non-human primates models, and gene therapy to combat virus infectious diseases, to correct monogenic disorders in vivo or in pluripotent cells. In this prospect article, after highlighting recent developments of CRISPR systems, we outline different applications and current limitations of CRISPR use in biological and biomedical investigation. Finally, we provide a perspective for future development and potential risks of this multifunctional technology.

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