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为青少年和成年期发病的亨廷顿舞蹈症开发干细胞疗法。

Developing stem cell therapies for juvenile and adult-onset Huntington's disease.

作者信息

Fink Kyle D, Deng Peter, Torrest Audrey, Stewart Heather, Pollock Kari, Gruenloh William, Annett Geralyn, Tempkin Teresa, Wheelock Vicki, Nolta Jan A

机构信息

Stem Cell Program & Institute for Regenerative Cures, University of California Davis Health Systems, 2921 Stockton Blvd. Sacramento, CA, USA 95817.

GenomeCenter, Biochemistry & Molecular Medicine, University of California, 451 Health Sciences Dr. Davis, CA 95616 USA.

出版信息

Regen Med. 2015;10(5):623-46. doi: 10.2217/rme.15.25.

Abstract

Stem cell therapies have been explored as a new avenue for the treatment of neurologic disease and damage within the CNS in part due to their native ability to mimic repair mechanisms in the brain. Mesenchymal stem cells have been of particular clinical interest due to their ability to release beneficial neurotrophic factors and their ability to foster a neuroprotective microenviroment. While early stem cell transplantation therapies have been fraught with technical and political concerns as well as limited clinical benefits, mesenchymal stem cell therapies have been shown to be clinically beneficial and derivable from nonembryonic, adult sources. The focus of this review will be on emerging and extant stem cell therapies for juvenile and adult-onset Huntington's disease.

摘要

干细胞疗法已被探索作为治疗中枢神经系统神经疾病和损伤的新途径,部分原因是它们具有模拟大脑修复机制的天然能力。间充质干细胞因其能够释放有益的神经营养因子以及营造神经保护微环境的能力而备受临床关注。虽然早期干细胞移植疗法充满技术和政治问题,且临床益处有限,但间充质干细胞疗法已被证明具有临床益处,并且可以从非胚胎、成人来源获得。本综述的重点将是针对青少年和成人发病的亨廷顿舞蹈病的新兴和现有干细胞疗法。

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