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Open Access Review

Translational research in Huntington’s disease: opening up for disease modifying treatment

Jean-Marc Burgunder

Author Affiliations

Swiss Huntington’s Disease Centre, Department of Neurology, University of Bern, Neurobu Clinics, Steinerstrasse 45, CH 3006, Bern, Switzerland

Departments of Neurology, West China Hospital, Sichuan University Chengdu, Xiangya Hospital, Central South University, and Changsha, Sun Yat Sen University, Guangzhou, China

Translational Neurodegeneration 2013, 2:2  doi:10.1186/2047-9158-2-2

Published: 25 January 2013

Abstract

Research on the molecular mechanisms involved in Huntington’s disease, a monogenic disorder with a complex phenotype including motor, behaviour, and cognitive impairments, is advancing at a rapid path. Knowledge on several of the multimodal pathways has now lead to the establishment of rational strategies to prepare trials of several compounds in affected people. Furthermore, improved understanding of the phenotype and on ways of assessing it, as well as the process of developing biomarkers, allows setting the frame for such studies. In this brief review, the present status of some of these aspects is examined.