Dopamine Cell-Based Replacement Therapies.

IF 7.8 2区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL
Saeed Kayhanian, Roger A Barker
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引用次数: 0

Abstract

Parkinson's disease (PD) is a common disorder that has, as part of its core pathology, the loss of the nigral dopaminergic nerve cells that project to the striatum. Replacing this loss with dopaminergic drugs has been the mainstay of therapy in PD for more than 50 years and while offering significant clinical benefit, especially in early-stage disease, leads to side effects over time. A conceptually more effective way to treat this aspect of the PD pathology would be to replace the missing dopaminergic system with grafts of new dopamine cells. This approach has been investigated for nearly 40 years using a variety of different dopamine cell sources. To date, a proof-of-principle has been shown using human fetal dopamine cells in patients with PD, but the more widespread adoption of this approach has been hampered by logistical reasons around tissue supply, the ethics of the cell source, and, most importantly, by the inconsistent results shown across trials, which in some cases have reported worrying side effects. Reasons for all this have been discussed extensively in the literature and one solution may lie in the development of new human stem cell-derived dopamine cells, which are now just entering first in human clinical trials.

基于多巴胺细胞的替代疗法。
帕金森病(PD)是一种常见疾病,其核心病理之一是投射到纹状体的黑质多巴胺能神经细胞丧失。50多年来,多巴胺能药物一直是治疗帕金森氏症的主要药物,虽然能带来显著的临床疗效,尤其是在疾病早期,但随着时间的推移会产生副作用。从概念上讲,治疗帕金森病病理的更有效方法是通过移植新的多巴胺细胞来替代缺失的多巴胺能系统。近40年来,人们一直在利用各种不同的多巴胺细胞来源研究这种方法。迄今为止,使用人类胎儿多巴胺细胞治疗帕金森病患者的原理已经得到证明,但由于组织供应的后勤原因、细胞来源的伦理问题,以及最重要的是,不同试验显示的结果并不一致,有些试验还报告了令人担忧的副作用,这种方法的广泛采用受到了阻碍。文献中已对所有这些原因进行了广泛讨论,其中一个解决方案可能是开发新的人类干细胞衍生多巴胺细胞。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
Cold Spring Harbor perspectives in medicine
Cold Spring Harbor perspectives in medicine MEDICINE, RESEARCH & EXPERIMENTAL-
CiteScore
14.30
自引率
1.90%
发文量
44
审稿时长
4-8 weeks
期刊介绍: Cold Spring Harbor Perspectives in Medicine is a monthly online publication comprising reviews on different aspects of a variety of diseases, covering everything from the molecular and cellular bases of disease to translational medicine and new therapeutic strategies. Cold Spring Harbor Perspectives in Medicine is thus unmatched in its depth of coverage and represents an essential source where readers can find informed surveys and critical discussion of advances in molecular medicine.
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