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

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 ...

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 ...

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.

This week, we heard an update from Roche about their huntingtin-lowering therapy, tominersen, currently being tested in the GENERATION HD2 trial. An independent data monitoring committee (iDMC) that ...

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 ...

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 ...

Huntingtin lowering has gained lots of attention in HD research, and for good reason. It was the first potential treatment designed to directly target the cause of HD – the huntingtin protein. But ...

Scientists often use genetics, the study of DNA, to understand the cellular changes that cause disease. By comparing people’s DNA with their symptoms, they can pinpoint specific genetic differences, ...

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 ...

AMT-130 is a huntingtin-lowering treatment, because it seeks to reduce production of the harmful mutant huntingtin protein, which is harmful to neurons and is the cause of Huntington’s disease. A ...