New Approach to Huntington's Disease Uncovered

Targeting a specific protein fragment may be more effective than current treatments

Mar. 19, 2026 at 5:08am

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A new study in mice suggests that treatments targeting a fragment of the mutant protein that causes Huntington's disease, called huntingtin 1a, may be more effective than current treatments that target the whole protein but leave this fragment intact.

Why it matters

Huntington's disease is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. Current experimental treatments aim to prevent production of the entire mutant protein, but this new research indicates that specifically targeting the huntingtin 1a fragment may be a more promising approach.

The details

The study found that treatments that blocked production of the whole mutant huntingtin protein had little effect, while treatments that prevented production of just the huntingtin 1a fragment were remarkably effective. This suggests that the huntingtin 1a fragment may be a key driver of the disease process and that future therapies should focus on targeting this specific component.

  • The findings were published on March 18, 2026 in the journal Science Translational Medicine.

The players

Jeffrey Carroll

An associate professor of neurology at the University of Washington School of Medicine in Seattle and the senior author of the study.

Robert Bragg

The study's first author and a research scientist in the Carroll laboratory.

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What they’re saying

“I hope we're wrong, but the science behind our findings is solid. To succeed, we may need to design new treatments that also target this specific region of the protein.”

— Jeffrey Carroll, Associate Professor of Neurology

“When I looked at the cells of the treated mice under the microscope, I thought I had made a mistake because at first I could find no protein aggregates. I had to look really hard to even find one or two.”

— Robert Bragg, Research Scientist

“It appears that if you lower the expression of the full Huntington's protein, but huntingtin 1a is still being expressed, it doesn't move the needle at all. It looks like you really need to lower huntingtin 1a to be effective.”

— Robert Bragg, Research Scientist

What’s next

The researchers plan to continue studying the role of the huntingtin 1a fragment and explore new treatments that specifically target this component of the mutant protein in order to develop more effective therapies for Huntington's disease.

The takeaway

This study suggests a promising new approach to treating Huntington's disease by targeting a specific fragment of the mutant protein, rather than the full protein, which could lead to more effective therapies for this devastating genetic disorder.