当前位置: X-MOL 学术Assay Drug Dev. Technol. › 论文详情
Our official English website, www.x-mol.net, welcomes your feedback! (Note: you will need to create a separate account there.)
The Promise and Perils of Compound Discovery Screening with Inducible Pluripotent Cell-Derived Neurons.
ASSAY and Drug Development Technologies ( IF 1.8 ) Pub Date : 2019-05-16 , DOI: 10.1089/adt.2019.914
Elizabeth R Sharlow 1, 2 , Mehmet Murat Koseoglu 1, 2 , George S Bloom 3, 4, 5 , John S Lazo 1, 2, 6, 7
Affiliation  

Neurological diseases comprise more than a thousand ailments that adversely affect the brain and nervous system. When grouped together, these neurological conditions impact an estimated 100 million individuals in the United States and up to a billion people worldwide, making drug discovery efforts imperative. However, recent research and development efforts for these neurological diseases, including Alzheimer's disease and amyotrophic lateral sclerosis, have been exceedingly disappointing and typify the challenges associated with translating in vitro and cell-based discoveries to successful preclinical models and subsequent human clinical trials. Our viewpoint is that neuronal progenitor cells and neurons derived from inducible pluripotent stem cells afford an innovative translational bridge, with higher pathological relevancy than previous cellular models. We outline some of the opportunities and challenges associated with their evolving usage in drug discovery and development.

中文翻译:

用可诱导的多能细胞衍生神经元进行化合物发现筛选的前景和危险。

神经系统疾病包括一千多种对大脑和神经系统产生不利影响的疾病。如果将这些神经疾病放在一起,那么在美国估计有1亿人受到影响,在全世界范围内有多达10亿人受到影响,因此药物研发工作势在必行。但是,针对这些神经系统疾病(包括阿尔茨海默氏病和肌萎缩性侧索硬化症)的最新研究和开发成果令人非常失望,并代表了将体外和基于细胞的发现转化为成功的临床前模型和随后的人类临床试验所面临的挑战。我们的观点是,神经元祖细胞和可诱导多能干细胞衍生的神经元提供了创新的翻译桥梁,具有比以前的细胞模型更高的病理相关性。我们概述了在药物研发中不断发展的应用所带来的一些机遇和挑战。
更新日期:2019-11-01
down
wechat
bug