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神经疾病的干细胞治疗翻译。

Translation of stem cell therapy for neurological diseases.

机构信息

Department of Neurology, University of Leipzig, Leipzig, Germany.

出版信息

Transl Res. 2010 Sep;156(3):155-60. doi: 10.1016/j.trsl.2010.07.002. Epub 2010 Jul 23.

Abstract

"Regenerative medicine" hopefully will provide novel therapies for diseases that remain without effective therapy. This development is also true for most neurodegenerative disorders including Alzheimer's disease, Huntington's disease, or Parkinson's disease. Transplantation of new neurons to the brain has been performed in Parkinson's disease and in Huntington's disease. The restoration of dopaminergic neurons in patients with Parkinson's disease via implantation of embryonic midbrain tissue was taken from animal experiments to clinical applications, showing a limited efficacy. Clinical trials in patients with Huntington's disease using fetal striatal tissue currently are underway. Today, it seems possible to generate functional dopaminergic or striatal neurons form a variety of stem cells including embryonic or neural stem cells as well as induced pluripotent stem cells. First clinical trials using neural stem cell or embryonic-stem-cell-derived tissue are approved or already underway. Such cells allow for extensive in vitro and in vivo testing as well as "good manufacturing production," reducing the risks in clinical application.

摘要

“再生医学”有望为那些目前尚无有效治疗方法的疾病提供新的治疗方法。这一发展也适用于大多数神经退行性疾病,包括阿尔茨海默病、亨廷顿病或帕金森病。已经在帕金森病和亨廷顿病中进行了向大脑中移植新神经元的治疗。通过植入胚胎中脑组织来恢复帕金森病患者的多巴胺能神经元,是从动物实验到临床应用的,显示出有限的疗效。目前正在进行使用胎儿纹状体组织治疗亨廷顿病的临床试验。如今,似乎可以从各种干细胞中生成功能性多巴胺能或纹状体神经元,包括胚胎干细胞或神经干细胞以及诱导多能干细胞。使用神经干细胞或胚胎干细胞衍生组织的首次临床试验已经获得批准或正在进行中。这些细胞允许进行广泛的体外和体内测试以及“良好的生产规范”,从而降低临床应用的风险。

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