, Volume 1, Issue 1, pp 128–138

The paradigm of Huntington’s disease: Therapeutic opportunities in neurodegeneration


DOI: 10.1602/neurorx.1.1.128

Cite this article as:
Leegwater-Kim, J. & Cha, JH.J. Neurotherapeutics (2004) 1: 128. 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 theHD 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.

Key Words

Huntington’s disease apoptosis excitotoxicity mitochondria transcription transplantation 

Copyright information

© The American Society for Experimental NeuroTherapeutics, Inc 2004

Authors and Affiliations

  1. 1.Department of NeurologyMassachusetts General HospitalCharlestown
  2. 2.MassGeneral Institute for Neurodegenerative Disease, Department of NeurologyMassachusetts General Hospital/B 114-2000Charlestown

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