title: An update on Huntington's disease: from the gene to the clinic.
abstract: OBJECTIVE
  This review highlights the recent advances in Huntington's disease, with a particular focus on development of disease biomarkers for use in therapeutic trials in the premotor phase of the disease, as well as the growing literature regarding pathophysiological mechanisms and their relevance to potential therapeutic targets.
  
  
  RESULTS
  There have been continued advances in the development of disease biomarkers, and promising neuroprotection trials are beginning to emerge in the premotor stage of Huntington's disease. Deeper understanding of the pathophysiological mechanisms is being translated into potential therapeutic strategies.
  
  
  CONCLUSIONS
  The premotor stage of Huntington's disease provides an ideal time to trial disease-modifying therapy, but reliable biomarkers are required for monitoring disease progression, and this remains an area of intense research. Our understanding of the underlying pathophysiological mechanisms continues to expand, and a number of promising therapeutic strategies are emerging, including strategies to silence mutant huntingtin expression.
authors: aMovement Disorders Unit, Department of Neurology, Westmead Hospital bSydney Medical School-Western, University of Sydney, Sydney, Australia.
authors: Samuel D
authors: SD
authors: Kim
authors: Victor S C
authors: VS
authors: Fung
mesh_terms: D006801:Humans; D006816:Huntington Disease / Q000235:genetics / Q000503:physiopathology / Q000628:therapy