Therapeutic prospects for the prevention of neurodegeneration in Huntington's disease and the polyglutamine repeat disorders.
Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by expansion of a polyglutamine (polyQ) tract in the huntingtin protein, resulting in intracellular aggregate formation and neurodegeneration. Biochemical pathways leading from polyQ expansion to disease pathogenesis are largely unknown. Recent approaches using genetic models systems have begun to uncover nuclear and cytoplasmic pathologies that represent potential targets for therapeutic intervention.
Kimura
Yoko
Y
Picower Institute for Learning and Memory, Massachusetts Institute of Technology, 46-3243, 43 Vassar St. Cambridge, MA 02139, USA. ykimura@mit.edu
Lee
Wyan-Ching Mimi
WC
Littleton
J Troy
JT
D006801:Humans; D006816:Huntington Disease / Q000188:drug therapy* / Q000378:metabolism / Q000473:pathology / Q000517:prevention & control*; D010455:Peptides / Q000378:metabolism*
D010455:Peptides; C097188:polyglutamine