利用猴模型开展神经疾病细胞及基因治疗的研究进展

彭 静,周 勤,陈永昌

神经病学与神经康复学杂志 ›› 2020, Vol. 16 ›› Issue (1) : 11-18.

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神经病学与神经康复学杂志 ›› 2020, Vol. 16 ›› Issue (1) : 11-18. DOI: 10.12022/jnnr.2019-0091
神经修复与神经免疫专题

利用猴模型开展神经疾病细胞及基因治疗的研究进展

  • 彭 静1,周 勤2,陈永昌2
作者信息 +

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

  • PENG Jing1, ZHOU Qin2, CHEN Yongchang2
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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。

Abstract

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 preclinical 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

Key words

Non-human primates  /  Neurodegenerative diseases  /  Neurogenetic diseases  /  Gene therapy  /  Cell
replacement therapy

引用本文

导出引用
彭 静,周 勤,陈永昌. 利用猴模型开展神经疾病细胞及基因治疗的研究进展[J]. 神经病学与神经康复学杂志. 2020, 16(1): 11-18 https://doi.org/10.12022/jnnr.2019-0091
PENG Jing, ZHOU Qin, CHEN Yongchang. Advances in the use of monkey models for neurological disease cells and gene therapy[J]. Journal of Neurology and Neurorehabilitation. 2020, 16(1): 11-18 https://doi.org/10.12022/jnnr.2019-0091

基金

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

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