Article
Huntingtin-lowering strategies for Huntington's disease.
Expert opinion on investigational drugs - 1 Oct 2020
Barker Roger A, Fujimaki Motoki, Rogers Priya, Rubinsztein David C
Abstract excerpt
INTRODUCTION: Huntington's disease (HD) is an incurable, autosomal dominant neurodegenerative disease caused by an abnormally long polyglutamine tract in the huntingtin protein. Because this mutation causes disease via gain-of-function, lowering huntingtin levels represents a rational therapeutic strategy. AREAS COVERED: We searched MEDLINE, CENTRAL, and other trial databases, and relevant company and HD funding...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
