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Huntington's disease is caused by repetitive "stutters" in DNA—specifically repeated CAG sequences—that expand abnormally over time, leading to toxic protein accumulation that contributes to the progression of the disease. There is currently no cure for this rare and fatal genetic disease.

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."

"We wanted to finesse this technique to explore whether there are more reliable potential genetic treatments for Huntington's disease."

In the new study, published in Science Translational Medicine, researchers used a more precise gene-editing enzyme called Cas9 nickase, which cuts only a single strand of DNA within the repeated CAG/CTG sequences in mouse models of Huntington's disease.

Professor Dion said, "When packaged into viral vectors and delivered directly to the striatum—the brain region primarily impacted by Huntington's disease—in a mouse model, the therapy shortened the DNA repeats in the majority of targeted neurons.

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