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用于神经退行性疾病的干细胞疗法:转分化问题

Stem cell therapy for neurodegenerative diseases: the issue of transdifferentiation.

作者信息

Cova Lidia, Ratti Antonia, Volta Manuela, Fogh Isabella, Cardin Veronica, Corbo Massimo, Silani Vincenzo

机构信息

Department of Neurology and Laboratory of Neuroscience, Dino Ferrari Center, University of Milan Medical School, IRCCS Istituto Auxologico Italiano, Milan, Italy.

出版信息

Stem Cells Dev. 2004 Feb;13(1):121-31. doi: 10.1089/154732804773099326.

Abstract

In the past few years research on stem cells has exploded as a tool to develop potential therapies to treat incurable neurodegenerative diseases. Stem cell transplantation has been effective in several animal models, but the underlying restorative mechanisms are still unknown. Several events such as cell fusion, neurotrophic factor release, endogenous stem cell proliferation, and transdifferentiation (adult cell acquisition of new unexpected identities) may explain therapeutic success, in addition to replacement of lost cells. This issue needs to be clarified further to maximize the potential for effective therapies. Preliminary stem transplantation trials have already been performed for some neurodegenerative diseases. There is no effective pharmacological treatment for amyotrophic lateral sclerosis, but recent preliminary data both in experimental and clinical settings have targeted it as an ideal candidate disease for the development of stem cell therapy in humans. This review summarizes recent advances gained in stem cell research applied to neurodegenerative diseases with a special emphasis to the criticisms put forward.

摘要

在过去几年中,干细胞研究呈爆发式增长,成为开发潜在疗法以治疗无法治愈的神经退行性疾病的一种工具。干细胞移植在多种动物模型中已见成效,但其潜在的修复机制仍不明晰。除了丢失细胞的替代作用外,诸如细胞融合、神经营养因子释放、内源性干细胞增殖以及转分化(成体细胞获得新的意外特性)等几种情况或许可以解释治疗成功的原因。为了将有效疗法的潜力最大化,这一问题需要进一步阐明。针对某些神经退行性疾病已经开展了初步的干细胞移植试验。肌萎缩侧索硬化症尚无有效的药物治疗方法,但近期实验和临床环境中的初步数据已将其作为人类干细胞治疗开发的理想候选疾病。本综述总结了干细胞研究应用于神经退行性疾病所取得的最新进展,并特别强调了所提出的批评意见。

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