Pluripotent stem cell-based therapy for Parkinson's disease: Current status and future prospects.
Pluripotent stem cell-based therapy for Parkinson's disease: Current status and future prospects.
复制标题
DOI：
10.1016/j.pneurobio.2018.04.005
复制
发表时间：
2018-09
影响因子：
6.7
通讯作者：
Kim KS
中科院分区：
文献类型：
--
作者：
Sonntag KC;Song B;Lee N;Jung JH;Cha Y;Leblanc P;Neff C;Kong SW;Carter BS;Schweitzer J;Kim KS
帕金森病（PD）是最常见的神经退行性疾病之一，影响约0.3%的普通人群。随着发达国家人口的老龄化，这在经济方面和这些患者及其家庭的生活质量方面给社会造成了不断增加的负担。虽然目前可用的药物或手术治疗可能会显着改善许多PD患者的生活质量，但这些都是对症治疗，不会减缓或阻止疾病的进展过程。由于帕金森病的运动障碍主要是由于黑质致密部中中脑多巴胺神经元的丢失造成的，因此帕金森病长期以来一直被认为是基于细胞治疗的最有希望的靶疾病之一。事实上，许多使用胎儿细胞移植的临床和临床前研究已经提供了概念证据，证明细胞替代疗法可能是PD的可行治疗方法。然而，使用人类胎儿细胞作为标准化治疗方案充满了基本的伦理，实践和临床问题，促使科学家探索替代细胞来源。基于人类胚胎干细胞和诱导多能干细胞的开创性建立，这些人类多能干细胞一直是广泛研究的主题，导致我们对这些新型干细胞的理解取得了巨大进步，并为再生医学带来了巨大潜力。在这篇综述中，我们讨论了人类多能干细胞为基础的细胞治疗PD的前景和挑战。
Parkinson’s disease (PD) is one of the most common neurodegenerative disorders, which affects about 0.3% of the general population. As the population in the developed world ages, this creates an escalating burden on society both in economic terms and in quality of life for these patients and for the families that support them. Although currently available pharmacological or surgical treatments may significantly improve the quality of life of many patients with PD, these are symptomatic treatments that do not slow or stop the progressive course of the disease. Because motor impairments in PD largely result from loss of midbrain dopamine neurons in the substantia nigra pars compacta, PD has long been considered to be one of the most promising target diseases for cell-based therapy. Indeed, numerous clinical and preclinical studies using fetal cell transplantation have provided proof of concept that cell replacement therapy may be a viable therapeutic approach for PD. However, the use of human fetal cells as a standardized therapeutic regimen has been fraught with fundamental ethical, practical, and clinical issues, prompting scientists to explore alternative cell sources. Based on groundbreaking establishments of human embryonic stem cells and induced pluripotent stem cells, these human pluripotent stem cells have been the subject of extensive research, leading to tremendous advancement in our understanding of these novel classes of stem cells and promising great potential for regenerative medicine. In this review, we discuss the prospects and challenges of human pluripotent stem cell-based cell therapy for PD.
登录
查看更多内容
DOI：
10.3233/jpd-160798
发表时间：
2016
期刊：
Journal of Parkinson's disease
影响因子：
--
作者：
Barker RA;Parmar M;Kirkeby A;Björklund A;Thompson L;Brundin P
通讯作者：
Brundin P
影响因子：
4.8
作者：
Allan, Laura E.;Petit, Geraldine H.;Brundin, Patrik
通讯作者：
Brundin, Patrik
影响因子：
16.6
作者：
Bhutani K;Nazor KL;Williams R;Tran H;Dai H;Džakula Ž;Cho EH;Pang AWC;Rao M;Cao H;Schork NJ;Loring JF
通讯作者：
Loring JF
影响因子：
4
作者：
Barzilay, Ran;Ben-Zur, Tali;Offen, Daniel
通讯作者：
Offen, Daniel
影响因子：
23.9
作者：
Ben-David, Uri;Gan, Qing-Fen;Benvenisty, Nissim
通讯作者：
Benvenisty, Nissim