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[基因与基因细胞治疗及神经退行性疾病]

[Gene and gene cellular therapy and neurodegenerative diseases].

作者信息

Ugriumov M V, Ermakov A S, Popov A P, Zhdanov R I

机构信息

Koltzov Institute of Developmental Biology RAS, Russia, Moscow.

出版信息

Vopr Med Khim. 2000 May-Jun;46(3):311-23.

Abstract

The modern methods of the treatment of the neurodegenerative diseases are considered. Neurodegenerative diseases originate due to the degeneration of the neuronal cells of central nervous system that leads to imbalance of the neurotransmitter synthesis and, as a consequence, movement disorders and mental disabilities. Traditional methods of pharmacotherapy and neurosurgery give short-term effect. Since 1980 neurotransplantation was developed as a new technology for the treatment of the neurodegenerative diseases. This approach represents a case of cell therapy being used for transplantation of the human fetal material. Cell transplantation compensates the local deficiency of the neurotransmitter level by substitution of degenerated neurons of patient's brain (e.g. dopaminergic neurons). Gene-cell conjunction of cell therapy with modification of genome of transplanted cells is the most perspective approach to increase an efficiency of neurotransplantation. Short description of gene therapy approaches and a search for optimal gene-cell protocols for therapy of neurodegenerative diseases are presented in this paper.

摘要

本文探讨了神经退行性疾病的现代治疗方法。神经退行性疾病是由于中枢神经系统神经元细胞退化所致,这会导致神经递质合成失衡,进而引发运动障碍和智力残疾。传统的药物治疗和神经外科手术方法只能产生短期效果。自1980年以来,神经移植作为一种治疗神经退行性疾病的新技术得以发展。这种方法是细胞治疗用于人类胎儿材料移植的一个实例。细胞移植通过替代患者大脑中退化的神经元(如多巴胺能神经元)来补偿神经递质水平的局部不足。细胞治疗与移植细胞基因组修饰的基因-细胞结合是提高神经移植效率最具前景的方法。本文介绍了基因治疗方法的简要描述以及寻找治疗神经退行性疾病的最佳基因-细胞方案。

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