期刊
NATURE REVIEWS NEUROLOGY
卷 11, 期 6, 页码 339-350出版社
NATURE PUBLISHING GROUP
DOI: 10.1038/nrneurol.2015.79
关键词
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资金
- American Brain and Parkinson's Disease Foundations Clinician-Scientist Development Award
- Harvard NeuroDiscovery Center
- Multiple System Atrophy Coalition
- National Research Service Award fellowship [F32NS061419]
- Thome Memorial Foundation
- National Institute on Aging
- Howard Hughes Medical Institute
- JPB Foundation
- Eleanor Schwartz Charitable Foundation
In the absence of a single preventive or disease-modifying strategy, neurodegenerative diseases are becoming increasingly prevalent in our ageing population. The mechanisms underlying neurodegeneration are poorly understood, making the target-based drug screening strategies that are employed by the pharmaceutical industry fraught with difficulty. However, phenotypic screening in neurons and glia derived from patients is now conceivable through unprecedented developments in reprogramming, transdifferentiation, and genome editing. We outline progress in this nascent field, but also consider the formidable hurdles to identifying robust, disease-relevant and screenable cellular phenotypes in patient-derived cells. We illustrate how analysis in the simple baker's yeast cell Saccharaomyces cerevisiae is driving discovery in patient-derived neurons, and how approaches in this model organism can establish a paradigm to guide the development of stem cell-based phenotypic screens.
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