Overview

Huntington's disease (HD) is an autosomal neurodegenerative disorder characterized by motor, behavioral, cognitive and metabolic dysfunction caused by the expansion of a CAG trinucleotide repeat region in the huntingtin gene (HTT).

Charles River has a long-standing track record for providing HD lead compound efficacy screening services. Fully customized studies with two internally validated mouse models, the transgenic R6/2 mouse and the Q175 knock-in mouse, capture all aspects of efficacy screening.

The models are accompanied by behavior and cognitive testing, magnetic resonance imaging (MRI) and magnetic resonance spectroscopy (MRS). In addition, several relevant biomarkers, such as immunohistochemical measurements and qPCR, can be included to further expand the platform.