Drug screening for Huntington's disease and other neurodegenerative disorders.
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 Huntington's 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.
Varma
Hemant
H
Department of Biological Sciences, Columbia University, 614 Fairchild Building, New York, NY 10027, USA. hv2108@columbia.edu
D000818:Animals; D017209:Apoptosis; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical; D057166:High-Throughput Screening Assays; D006816:Huntington Disease / Q000188:drug therapy*; D019636:Neurodegenerative Diseases / Q000188:drug therapy*; D011499:Protein Processing, Post-Translational; D011506:Proteins / Q000737:chemistry / Q000378:metabolism / Q000633:toxicity; D017382:Reactive Oxygen Species / Q000378:metabolism; D054852:Small Molecule Libraries / Q000737:chemistry* / Q000627:therapeutic use
D011506:Proteins; D017382:Reactive Oxygen Species; D054852:Small Molecule Libraries