Antao Ainsley Mike, Karapurkar Janardhan Keshav, Lee Dong Ryul, Kim Kye-Seong, Ramakrishna Suresh
Graduate School of Biomedical Science and Engineering, Hanyang University, Seoul, South Korea.
Department of Biomedical Science, College of Life Science, CHA University, Seoul, South Korea.
Comput Struct Biotechnol J. 2020 Nov 19;18:3649-3665. doi: 10.1016/j.csbj.2020.11.026. eCollection 2020.
CRISPR/Cas systems are popular genome editing tools that belong to a class of programmable nucleases and have enabled tremendous progress in the field of regenerative medicine. We here outline the structural and molecular frameworks of the well-characterized type II CRISPR system and several computational tools intended to facilitate experimental designs. The use of CRISPR tools to generate disease models has advanced research into the molecular aspects of disease conditions, including unraveling the molecular basis of immune rejection. Advances in regenerative medicine have been hindered by major histocompatibility complex-human leukocyte antigen (HLA) genes, which pose a major barrier to cell- or tissue-based transplantation. Based on progress in CRISPR, including in recent clinical trials, we hypothesize that the generation of universal donor immune-engineered stem cells is now a realistic approach to tackling a multitude of disease conditions.
CRISPR/Cas系统是广受欢迎的基因组编辑工具，属于一类可编程核酸酶，在再生医学领域取得了巨大进展。我们在此概述了特征明确的II型CRISPR系统的结构和分子框架以及旨在促进实验设计的几种计算工具。使用CRISPR工具生成疾病模型推动了对疾病状况分子层面的研究，包括揭示免疫排斥的分子基础。主要组织相容性复合体-人类白细胞抗原（HLA）基因阻碍了再生医学的发展，该基因对基于细胞或组织的移植构成了重大障碍。基于CRISPR的进展，包括近期的临床试验，我们推测生成通用供体免疫工程干细胞现在是应对多种疾病状况的现实方法。
