title: Preclinical models: needed in translation? A Pro/Con debate.
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.
authors.affiliation: Johns Hopkins University, Brain Science Institute, Baltimore, Maryland, USA.
authors.forename: Thomas
authors.initials: T
authors.lastname: Philips
authors.forename: Jeffrey D
authors.initials: JD
authors.lastname: Rothstein
authors.forename: Mahmoud A
authors.initials: MA
authors.lastname: Pouladi
mesh_terms: D000818:Animals; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical; D006801:Humans; D006816:Huntington Disease / Q000235:genetics / Q000628:therapy*; D057170:Translational Research, Biomedical / Q000379:methods*
keywords: Animal models; HTT; Huntington's; transgenic; translation