The potential of gene editing for Huntington's disease.

Abstract excerpt

Huntington's disease (HD) is a dominantly inherited neurodegenerative disorder caused by a trinucleotide repeat expansion in the huntingtin gene resulting in long stretches of polyglutamine repeats in the huntingtin protein. The disease involves progressive degeneration of neurons in the striatum and cerebral cortex resulting in loss of control of motor function, psychiatric problems, and cognitive deficits....

Topics

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

• Share to CRISPR-Cas Systems
• Share to Disease Models, Animal
• Share to Gene Editing