《生命科学》 2020, 32(7): 669-675

基因编辑猴模型及其在基因治疗研究中的应用前景

肖浦豪 , 张　波 , 陈永昌^*

昆明理工大学灵长类转化医学研究院，云南中科灵长类生物医学重点实验室，昆明 650500

摘　要：

基因编辑技术的开创性进展使得利用CRISPR 技术建立猴模型并开展病变基因校正成为目前基因治疗研究领域的热点。非人灵长类与人类在进化关系上最为接近，在模型构建、疾病机制研究以及药物研发方面优势突出。随着基因修饰技术在非人灵长类上的逐步应用，目前已经构建出多种与临床患者病症高度吻合的疾病模型，为开展遗传疾病的基因治疗打下了坚实基础。然而，目前基因递送和基因修复系统面临巨大挑战，能否安全、高效、精确地修复致病基因是基因治疗临床转化的关键问题，现将综述基于猴模型开展基因治疗研究的前景及挑战。

通讯作者：陈永昌 , Email:chenyc@lpbr.cn

Gene editing monkey model and its application prospect in gene therapy research

XIAO Pu-Hao , ZHANG Bo , CHEN Yong-Chang^*

Yunnan Key Laboratory of Primate Biomedical Research, Institute of Primate Translational Medicine, Kunming University of Science and Technology, Kunming 650500, China

Abstract:

The pioneering advances in gene editing have led the establishment of monkey models and the correction of disease genes using the CRISPR system to become a hot topic in current gene therapy research. Non-human primates have the closest evolutionary relationship with humans, and have prominent advantages in model building, disease mechanism research and drug development. With the gradual application of gene modification technology in non-human primates, a variety of disease models that are highly consistent with clinical patients have been
constructed, laying a solid foundation for gene therapy of genetic diseases. However, current gene delivery and gene repair systems face enormous challenges. The ability to safely, efficiently and accurately repair pathogenic genes is a key issue in the clinical transformation of gene therapy. This article will review the prospects and challenges of gene therapy research based on the monkey model.

Communication Author：CHEN Yong-Chang , Email:chenyc@lpbr.cn