Preclinical models: Needed in translation? A Pro/Con debate

Thomas Philips, Jeffrey D. Rothstein, Mahmoud A. Pouladi

Research output: Contribution to journalArticlepeer-review

9 Scopus citations


The discovery of the causative mutations and many of the predisposing risk factors for neurodegenerative disorders such as Amyotrophic Lateral Sclerosis, Alzheimer's, Parkinson's, and Huntington's disease (HD), has led to the development of a large number of genetic animal models of disease. In the case of HD, for example, over 20 different transgenic rodent models have been generated. These models have been of immense value in providing novel insights into mechanisms of disease, with the promise of accelerating the development of therapies that can delay the onset or slow the progression of the disease. Yet, despite extensive use of such models, no effective treatment for HD has been developed. Here, we discuss the value of animal models, highlighting their strengths and shortcomings in the context of translational research for HD.

Original languageEnglish (US)
Pages (from-to)1391-1396
Number of pages6
JournalMovement Disorders
Issue number11
StatePublished - Sep 15 2014


  • Animal models
  • HTT
  • Huntington's
  • Transgenic
  • Translation

ASJC Scopus subject areas

  • Neurology
  • Clinical Neurology


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