An insight into allele-selective approaches to lowering mutant huntingtin protein for Huntington's disease treatment.

Abstract excerpt

Huntington's disease (HD), a monogenic neurodegenerative disorder, stems from a CAG repeat expansion within the mutant huntingtin gene (HTT). This leads to a detrimental gain-of-function of the mutated huntingtin protein (mHTT). As of now, there exist no efficacious therapies to alter the disease progression. In view of the monogenetic mutation nature and an indispensable role of wild-type HTT in healthy...

Topics

• Huntington Disease
• Huntingtin Protein
• Humans
• Animals
• Alleles
• Mutation
• Genetic Therapy