Targeting mutant huntingtin for the development of disease-modifying therapy.

Abstract excerpt

Huntington's disease (HD) is a progressive and fatal neurodegenerative disease, and the most common inherited CAG repeat disorder. A polyglutamine expansion in the N-terminus of the huntingtin protein (HTT) leads to protein misfolding and downstream pathogenic processes culminating in widespread functional impairment and neurodegeneration in the striatum, cortex and other brain areas. To date, only symptomatic...

Topics

Share this publication in a Topic to start or enrich a Post.

• Share to Brain
• Share to Cognition Disorders
• Share to Drug Design