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Developing Stem Cell Therapies for Parkinson's Disease: Waiting Until the Time Is Right

机译:发展针对帕金森氏病的干细胞疗法:等到时机成熟

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The ability to successfully replace lost nigral dopaminergic neurons in Parkinson's disease (PD) has been clearly shown with fetal ventral mesencephalic transplants, albeit inconsistently. The need to trial this approach with stem-cell-derived neurons is approaching, but it should only commence when all the key issues have been adequately resolved. Parkinson's disease (PD) is a common neurodegenerative disorder that affects about 1 in 800 people worldwide and is currently incurable. It is characterized by a movement disorder associated with the loss of the nigral dopaminergic neurons, and while it is now well described that the pathology and clinical features extend outside these domains, it can nevertheless be successfully treated for many years with dopaminergic drugs. However, over time these drugs become less effective as the disease progresses and generate their own unique side effects such as dyskine-sias. As such over the last 30 years there have many attempts to better deliver dopa-mine to the parkinsonian brain using cell-based therapies and many lessons have been learned from these studies.
机译:帕金森氏病(PD)已成功显示出成功替代帕金森氏病(PD)中失去的黑质多巴胺能神经元的能力,尽管不一致。对干细胞源性神经元尝试这种方法的需求日益迫切,但是只有在所有关键问题都得到充分解决后,这种方法才应开始。帕金森氏病(PD)是一种常见的神经退行性疾病,全世界约有80​​0人患有帕金森氏病,目前尚无法治愈。它的特征是与黑质多巴胺能神经元丢失有关的运动障碍,尽管现在已经很好地描述了病理学和临床特征超出了这些领域,但仍可以用多巴胺能药物成功治疗多年。但是,随着时间的流逝,这些药物随着疾病的发展而变得无效,并会产生自身独特的副作用,例如运动障碍。因此,在过去的30年中,人们进行了许多尝试,以基于细胞的疗法将多巴胺更好地递送至帕金森氏脑,并且从这些研究中学到了很多教训。

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