Huntington disease: new insights into molecular pathogenesis and therapeutic opportunities.

Abstract excerpt

Huntington disease (HD) is a neurodegenerative disease caused by CAG repeat expansion in the huntingtin gene (HTT) and involves a complex web of pathogenic mechanisms. Mutant HTT (mHTT) disrupts transcription, interferes with immune and mitochondrial function, and is aberrantly modified post-translationally. Evidence suggests that the mHTT RNA is toxic, and at the DNA level, somatic CAG repeat expansion in...

Topics

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

• Share to Biomarkers
• Share to Clinical Trials as Topic
• Share to DNA Repair