Movement Disorders Foundation

A new study suggests that targeting a toxic genetic fragment called HTT1a may offer a more effective treatment strategy for Huntington’s disease than approaches aimed only at the full-length huntingtin protein. Researchers used small interfering RNA (siRNA), a technology designed to break down harmful RNA messages before toxic proteins can form, in mouse models of the disease.

The study found that lowering HTT1a reduced toxic protein clumping in the brain, delayed disease-related genetic changes, and improved abnormal gene activity more effectively than targeting full-length HTT alone. Benefits were strongest when treatment was started early, particularly in the hippocampus, a brain region involved in memory and emotion. Mice receiving repeated early treatment showed the greatest reduction in harmful protein aggregates.

The findings suggest that HTT1a plays a major role in disease progression and may represent an important target for future therapies. Researchers say future treatments may work best by targeting both HTT and HTT1a, or HTT1a specifically. Click https://en.hdbuzz.net/fixing-the-recipe-lowering-a-slice-of-huntingtin/ to learn more.