Professor Vincent Dion of the UK Dementia Research Institute at Cardiff University, who led the research team, said, “The length of these repeated sequences accounts for most of the variation in how severe the disease is, so targeting and reducing these sequences is a potentially viable therapeutic avenue.
“Traditional gene-editing techniques often cut both strands of DNA to disable mutated genes, but this can trigger unpredictable rearrangements, off-target mutations or damage to healthy copies of the gene. Some researchers are also trying to apply these traditional gene-editing techniques to treating Huntington’s disease in preclinical work, but they have limitations.”