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Stem cell-based therapy for Huntington's disease

机译：基于干细胞的亨廷顿氏病治疗

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摘要

Huntington's disease (HD) is a late-onset neurodegenerative disease characterized by a progressive loss of medium spiny neurons in the basal ganglia. The development of stem cell-based therapies for HD aims to replace lost neurons and/or to prevent cell death. This review will discuss pre-clinical studies which have utilized stem or progenitor cells for transplantation therapy using HD animal models. In several studies, neural stem and progenitor cells used as allotransplants and xenografts have been shown to be capable of surviving transplantation and differentiating into mature GABAergic neurons, resulting in behavioral improvements. Beneficial effects have also been reported for transplantation of stem cells derived from non-neural tissue, for example, mesenchymal- and adipose-derived stem cells, which have mainly been attributed to their secretion of growth and neurotrophic factors. Finally, we review studies using stem cells genetically engineered to over-express defined neurotrophic factors. While these studies prove the potential of stem cells for transplantation therapy in HD, it also becomes clear that technical and ethical issues regarding the availability of stem cells must be solved before human trials can be conducted. J. Cell. Biochem. 114: 754-763, 2013. ? 2012 Wiley Periodicals, Inc.
机译：亨廷顿舞蹈病（HD）是一种迟发性神经退行性疾病，其特征是基底神经节中突棘神经元逐渐丧失。 HD干细胞疗法的发展旨在替代失去的神经元和/或预防细胞死亡。这篇综述将讨论临床前研究，这些研究已经利用干细胞或祖细胞进行了高清动物模型的移植治疗。在一些研究中，已证明用作同种异体移植和异种移植的神经干细胞和祖细胞能够存活下来，并能够分化为成熟的GABA能神经元，从而改善行为。对于非神经组织来源的干细胞，例如间充质和脂肪干细胞的移植，也有有益的作用，这主要归因于它们的生长分泌和神经营养因子。最后，我们回顾了使用经过基因工程改造的干细胞来过度表达确定的神经营养因子的研究。这些研究证明了干细胞在HD移植治疗中的潜力，但同时也很清楚，在进行人体试验之前，必须解决与干细胞可用性有关的技术和伦理问题。 J.细胞。生化。 114：754-763，2013年。 2012 Wiley期刊公司

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