A commentary on the utility of a new L-DOPA-responsive dystonia mouse model. Issue 1 (1st January 2016)

• Record Type: Journal Article
• Authors: Rose, Samuel J.; Hess, Ellen J.
• Is Part Of: Rare diseases. Volume 4:Issue 1(2016)
• Publication Date: 2016-01-01
• Subjects: Basal ganglia -- dopamine -- neurogenetics -- striatum -- tyrosine hydroxylase; Rare diseases -- Periodicals; Rare Diseases; Rare diseases; Periodicals; Periodicals; 616
• DOI: 10.1080/21675511.2015.1128617
• Languages: English
• ISSNs: 2167-5511

Abstract

ABSTRACT: In a recent issue of Brain, we reported on the generation and characterization of a mouse model of the rare disease L-DOPA-responsive dystonia (DRD). Here, we discuss the utility of these mice for understanding broader disease processes and treatment strategies. Using specific experimental designs that either work "forward" from genetic etiology or "backward" from the symptomatic presentation, we discuss how our data and future work can be used to understand broader themes.