Scientists engineered stem cells with "interrupted" CAG repeats to break up the toxic stretch. This may stop expansion, and could improve problems in cells that model Huntington’s disease. This study suggests that DNA spelling can drive the disease.

POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.

CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New research finds that blocking cells from using their HTT gene slows this wear and tear, which might slow the onset of symptoms in HD.