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Rare Dis. 2014 Mar 31;2:e28637. doi: 10.4161/rdis.28637. eCollection 2014.

The challenge in translating basic research discoveries to treatment of Huntington disease.

Author information

  • 1Department of Chemical and Systems Biology; Stanford University School of Medicine; Stanford, CA USA.
  • 2Department of Physiology & Biophysics; Center of Mitochondrial Disease; Case Western Reserve University School of Medicine; Cleveland, OH USA.

Abstract

Huntington disease is a rare neurodegenerative disease resulting from insertion and/or expansion of a polyglutamine repeats close to the N-terminal of the huntingtin protein. Although unequivocal genetic tests have been available for about 20 years, current pharmacological treatments do not prevent or slow down disease progression. Recent basic research identified potential novel drug targets for the treatment of Huntington disease. However, there are clear challenges in translating these discoveries into treatment strategies for these patients. The following is a brief discussion of these challenges using our recent experience as an example.

KEYWORDS:

Drp1; Huntington disease; P110 peptide inhibitor; animal model; mitochondria; neurodegeneration; polyglutamine; protein-protein interactions

PMID:
25054095
PMCID:
PMC4091548
DOI:
10.4161/rdis.28637
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