利用猴模型开展神经疾病细胞及基因治疗的研究进展  

Advances in the use of monkey models for neurological disease cells and gene therapy

作　　者：彭静 周勤 陈永昌 PENG Jing;ZHOU Qin;CHEN Yongchang(Department of Neurology,Renji Hospital,Shanghai Jiao Tong University School of Medicine,Shanghai 200127,China;Institute of Primate Translational Medicine,Kunming University of Science and Technology/Yunnan Key Laboratory of Primate Biomedical Research,Kunming 650500,Yunnan Province,China)

机构地区：[1]上海交通大学医学院附属仁济医院神经内科,上海200127 [2]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,云南昆明650500

出　　处：《神经病学与神经康复学杂志》2020年第1期11-18,共8页Journal of Neurology and Neurorehabilitation

基　　金：国家重点研发计划(2018YFA0107902);国家自然科学基金(81930121)

摘　　要：非人灵长类动物与人类在生理构造、大脑结构与功能、生殖生理以及昼夜活动节律等方面均非常相似,因此其在神经系统疾病的机制研究和药物研发中具有显著而独特的优势。近年来,利用药物诱导、物理损伤以及基因工程技术,已逐步建立了多种神经系统疾病的猴模型,为开展细胞治疗、基因治疗以及新药研发提供了重要的实验模型。本文综述了神经系统疾病猴模型的特点以及在神经退行性疾病和遗传性神经发育疾病中的优势及其应用,阐述了利用猴模型开展神经疾病细胞及基因治疗的研究进展,期望对利用猴模型进一步探究疾病机制以及推进新疗法的临床前及临床研究提供参考。The physiological structure,brain structure and function,reproductive physiology and circadian rhythm of non-human primates are highly similar to those of humans.Therefore,primates model has significant and unique advantages in mechanism research and drug development of neurological diseases.In recent years,a variety of monkey models of neurological diseases has been gradually established using drug induction,physical injury and gene editing technology,providing an important experimental model for cell therapy,gene therapy and development of new drugs.In this review,the characteristics of monkey models and the advantages in cell and gene therapy of neurodegenerative diseases and inherited neurodevelopmental diseases are summarized,and the advances in the use of monkey models for neurological disease cells and gene therapy is reviewed,hoping to provide references for further investigation of disease mechanisms and advancement of pre-clinical and clinical research of new therapies using monkey models.

关 键 词：非人灵长类 神经退行性疾病 神经遗传性疾病 基因治疗 细胞替代治疗 

分 类 号：R741[医药卫生—神经病学与精神病学] R-332[医药卫生—临床医学]