Clinical Trials in a Dish: The Potential of Pluripotent Stem Cells to Develop Therapies for Neurodegenerative Diseases

被引：58

作者：

Haston, Kelly M. ^{[1
]}

Finkbeiner, Steven ^{[1
,2
,3
,4
,5
]}

机构：

[1] Gladstone Inst Neurol Dis, San Francisco, CA 94158 USA

[2] Taube Koret Ctr Neurodegenerat Dis, San Francisco, CA 94158 USA

[3] Hellman Family Fdn Program Alzheimers Dis Res, San Francisco, CA 94158 USA

[4] Univ Calif San Francisco, Dept Neurol, San Francisco, CA 94143 USA

[5] Univ Calif San Francisco, Dept Physiol, San Francisco, CA 94143 USA

来源：

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY, VOL 56 | 2016年 / 56卷

基金：

美国国家科学基金会;

关键词：

clinical trials; human pluripotent stem cells; drug development; neurodegeneration; AMYOTROPHIC-LATERAL-SCLEROSIS; OLIGODENDROCYTE PROGENITOR CELLS; ADULT HUMAN FIBROBLASTS; MOTOR-NEURONS; DIRECT CONVERSION; HIGH-THROUGHPUT; HUMAN ES; ALZHEIMERS-DISEASE; ANIMAL-MODELS; HUNTINGTONS-DISEASE;

D O I：

10.1146/annurev-pharmtox-010715-103548

中图分类号：

R9 [药学];

学科分类号：

1007 ;

摘要：

Neurodegenerative diseases are a leading cause of death. No disease-modifying therapies are available, and preclinical animal model data have routinely failed to translate into success for therapeutics. Induced pluripotent stem cell (iPSC) biology holds great promise for human in vitro disease modeling because these cells can give rise to any cell in the human brain and display phenotypes specific to neurodegenerative diseases previously identified in postmortem and clinical samples. Here, we explore the potential and caveats of iPSC technology as a platform for drug development and screening, and the future potential to use large cohorts of disease-bearing iPSCs to perform clinical trials in a dish.

引用

页码：489 / 510

页数：22

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