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 ...

CAG repeats can get longer over time as the HD gene is used, like the growing potholes and cracks in an old road. New ...

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.

Long repetitive sequences of C-A-G letters in the DNA code are associated with at least 12 genetic diseases, including Huntington’s disease (HD). A group of scientists in Massachusetts, USA, have ...

By setting up both reactions side by side in a test tube, the team revealed a literal tug-of-war between two DNA-repair pathways acting on the same HTT repeat. Which side wins likely determines ...

DNA-based drugs called antisense oligonucleotides, or ASOs, are now in multiple clinical trials in Huntington’s disease, aiming to lower production of the harmful mutant huntingtin protein in the ...

Multiple new studies have identified what may be the most important new fact about the genetics of Huntington’s disease since the gene was discovered in 1993. At least two research groups around the ...

UniQure has announced positive top-line results from its Phase I/II trial of AMT-130, a one-time gene therapy being tested in people with Huntington’s disease (HD). Topline data is a summary of the ...

A new CRISPR-based technology, called RIDE, is a leap forward for this trail-blazing technology. With the precision of a scalpel sharp enough to rewrite the very code of life, researchers have used it ...

HDBuzz: 2024 was a big year for HD research, particularly in the clinical space. Which breakthroughs or advances in HD research from the past year are you particularly excited about? HDBuzz Editors-in ...

A 14-year study tracking NfL levels in people with the HD gene shows this tiny protein can signal disease progression many years before symptoms start. The longest follow-up study yet adds weight to ...

On 25th June 2024, Wave Life Sciences shared the results of their SELECT-HD clinical trial. This trial tested a therapy called WVE-003, designed to only lower the expanded, harmful form of the ...