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

Thomas Philips, Jeffrey D Rothstein, Mahmoud A. Pouladi

Research output: Contribution to journalArticle

Abstract

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 : official journal of the Movement Disorder Society
Volume29
Issue number11
DOIs
StatePublished - Sep 15 2014

Fingerprint

Huntington Disease
Animal Models
Animal Diseases
Translational Medical Research
Genetic Models
Amyotrophic Lateral Sclerosis
Causality
Neurodegenerative Diseases
Parkinson Disease
Disease Progression
Rodentia
Alzheimer Disease
Mutation
Therapeutics

Keywords

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

ASJC Scopus subject areas

  • Clinical Neurology
  • Neurology
  • Medicine(all)

Cite this

Preclinical models : Needed in translation? A Pro/Con debate. / Philips, Thomas; Rothstein, Jeffrey D; Pouladi, Mahmoud A.

In: Movement disorders : official journal of the Movement Disorder Society, Vol. 29, No. 11, 15.09.2014, p. 1391-1396.

Research output: Contribution to journalArticle

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