Devising therapies for neurodegenerative diseases remains a major challenge due to the complex etiology, prolonged disease course and the paucity of validated targets. These factors make it difficult to model neurodegenerative diseases in a manner amenable to large-scale screening. However, recent developments in automation, combinatorial chemistry and high-throughput phenotypic assays have presented new opportunities for discovering small molecule therapeutics for neurodegenerative diseases. This review focuses on novel in vitro and phenotypic screens for Huntingtons disease and a few other neurodegenerative diseases. The lessons learned from these screens and the potential of the small molecules identified as therapeutic leads are discussed.
Keywords: Drug discovery, huntington's disease, models, polyglutamine disease, screening, therapy, Amyotrophic Lateral Sclerosis, ALS, SMER's, small molecule enhancers of rapamy, synuclein, FGF-2, CNTF, brain derived growth factor, BDNF, Fluorescence Resonance Energy transfer, FRET, Y-27632, spinocerebellar ataxia-1, SCA-1, su-peroxide dismutase, SOD, caffeic acid phenylethyl ester, CAPE
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