The paradigm of Huntington's disease: therapeutic opportunities in neurodegeneration

NeuroRx. 2004 Jan;1(1):128-38. doi: 10.1602/neurorx.1.1.128.


Despite a relatively small number of affected patients, Huntington's disease (HD) has been a historically important disease, embodying many of the major themes in modern neuroscience, including molecular genetics, selective neuronal vulnerability, excitotoxicity, mitochondrial dysfunction, apoptosis, and transcriptional dysregulation. The discovery of the HD gene in 1993 opened the door to the mechanisms of HD pathogenesis. Multiple pathologic mechanisms have been discovered, each one serving as a potential therapeutic target. HD thus continues to serve as a paradigmatic disorder, with basic bench research generating clinically relevant insights and stimulating the development of therapeutic human trials.

Publication types

  • Review

MeSH terms

  • Animals
  • Brain / pathology*
  • Brain / physiopathology*
  • Cell Transplantation
  • Humans
  • Huntington Disease / genetics
  • Huntington Disease / physiopathology*
  • Huntington Disease / therapy*
  • Nerve Degeneration / genetics
  • Nerve Degeneration / physiopathology
  • Nerve Degeneration / therapy
  • Neuroprotective Agents / therapeutic use*


  • Neuroprotective Agents