Huntington's disease: new hope for therapeutics.
Huntington's disease (HD) is one of eight progressive neurodegenerative disorders in which the underlying mutation is a CAG expansion encoding a polyglutamine tract. There are currently no cures or even effective therapies for HD. Effective strategies have remained elusive because little is known about either the mechanisms of expansion or the mechanism of polyglutamine-mediated neuronal death. However, recent advances in understanding the basic mechanisms of expansion and toxicity have renewed hope that a therapeutic strategy might someday be possible. Strategies effective in the treatment of HD are likely to be relevant in the treatment of a range of neurological and neurodegenerative disorders.
McMurray
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Dept Molecular Pharmacology and Experimental Therapeutics, Mayo Clinic and Foundation, Rochester, MN 55905, USA. mcmurray.cynthia@mayo.edu
D000818:Animals; D015316:Genetic Therapy / Q000639:trends*; D006801:Humans; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000235:genetics* / Q000628:therapy*; D009419:Nerve Tissue Proteins / Q000235:genetics; D009687:Nuclear Proteins / Q000235:genetics
C086055:HTT protein, human; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins