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Nat Rev Neurol. 2015 Jun;11(6):339-50. doi: 10.1038/nrneurol.2015.79. Epub 2015 May 19.

Toward stem cell-based phenotypic screens for neurodegenerative diseases.

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Department of Neurology, Massachusetts General Hospital and Harvard Medical School, WACC-835, 15 Parkman Street, Boston, MA 02114, USA.
Whitehead Institute for Biomedical Research, 9 Cambridge Center, Cambridge, MA 02142, USA.


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.

[Indexed for MEDLINE]

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