Therapeutic opportunities in polyglutamine disease.
Polyglutamine diseases comprise a class of familial neurodegenerative disorders caused by expression of proteins containing expanded polyglutamine tracts. Great progress has been made in elucidating the molecular mechanisms contributing to polyglutamine pathology, and in identifying potential drug targets. Although much remains to be learned, these advances provide an opportunity for rational approaches to target-based drug discovery.
Hughes
R E
RE
Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Washington, USA. rehughes@u.washington.edu
Olson
J M
JM
D000818:Animals; D017209:Apoptosis; D020169:Caspases / Q000378:metabolism; D015195:Drug Design; D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy / Q000235:genetics; D018832:Molecular Chaperones / Q000378:metabolism; D009410:Nerve Degeneration / Q000209:etiology; D019636:Neurodegenerative Diseases / Q000188:drug therapy* / Q000235:genetics*; D010455:Peptides / Q000235:genetics*; D017510:Protein Folding; D014158:Transcription, Genetic; D018911:Trinucleotide Repeats
D018832:Molecular Chaperones; D010455:Peptides; C097188:polyglutamine; D020169:Caspases