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Trends Biotechnol. 2017 Jul;35(7):625-639. doi: 10.1016/j.tibtech.2017.04.002. Epub 2017 May 25.

Optogenetic Approaches to Drug Discovery in Neuroscience and Beyond.

Author information

1
Department of Chemistry and Chemical Biology, Howard Hughes Medical Institute, Department of Physics, Harvard University, Cambridge, MA 02138, USA.
2
Department of Chemistry and Chemical Biology, Howard Hughes Medical Institute, Department of Physics, Harvard University, Cambridge, MA 02138, USA. Electronic address: cohen@chemistry.harvard.edu.

Abstract

Recent advances in optogenetics have opened new routes to drug discovery, particularly in neuroscience. Physiological cellular assays probe functional phenotypes that connect genomic data to patient health. Optogenetic tools, in particular tools for all-optical electrophysiology, now provide a means to probe cellular disease models with unprecedented throughput and information content. These techniques promise to identify functional phenotypes associated with disease states and to identify compounds that improve cellular function regardless of whether the compound acts directly on a target or through a bypass mechanism. This review discusses opportunities and unresolved challenges in applying optogenetic techniques throughout the discovery pipeline - from target identification and validation, to target-based and phenotypic screens, to clinical trials.

KEYWORDS:

drug discovery; neuroscience; optogenetics; optopatch; phenotypic screening

PMID:
28552428
PMCID:
PMC5495001
DOI:
10.1016/j.tibtech.2017.04.002
[Indexed for MEDLINE]
Free PMC Article

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