New gene-editing approach shows promise in treating Huntington's disease in mice
· Medical Xpressedited by Sadie Harley, reviewed by Robert Egan
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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."
Single-strand cuts shorten DNA repeats
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.
"This led to fewer toxic huntingtin protein aggregates, restored healthier gene expression pathways in brain cells and showed significant improvements in motor function during locomotion tests."
The team says no unwanted mutations were detected in brain cells in the mouse models with this approach, and it offers a potentially safer alternative to conventional gene-editing methods.
Further testing before human trials
"While much more research is needed—and our results only apply to mouse models of Huntington's—these are exciting findings because there is currently no treatment for Huntington's disease that changes the course of the disease. This could represent a novel therapeutic pathway through which we may be able to prevent the progression of—or effectively treat—Huntington's disease."
The research was based on the CRISPR gene-editing method and has potential application beyond Huntington's disease in other genetic diseases caused by expanded repetitions in genetic codes.
Now, the team says more research is needed to optimize delivery methods to ensure stable, long-term expression of the editing tools without triggering immune responses, investigate whether treating animals at earlier stages can prevent neurodegenerative damage before symptoms ever appear, and conduct rigorous safety and efficiency testing in larger preclinical models to build the foundation for human clinical trials.
Publication details
Alvaro Murillo et al., Cas9 nickase–mediated contraction of CAG/CTG repeats is associated with phenotypic improvements in a Huntington's disease mouse model, Science Translational Medicine (2026). DOI: 10.1126/scitranslmed.aef9466
Journal information: Science Translational Medicine
Key medical concepts
Huntington's DiseaseMotor function
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NeurologyClinical genetics Provided by Cardiff University Who's behind this story?
Sadie Harley
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