FDA grants “Investigational New Drug” status to Huntingtin-lowering gene-therapy agent AMT-130, clearing path to human trials in Huntington’s Disease patients
This fall sees exciting announcements from a number of companies focused on novel Huntingtin Lowering technologies, including Wave, PTC and Voyager
Completely removing normal huntingtin in adults may disrupt healthy brain function, a recent study suggests.
Work with genome editing techniques (zinc fingers and CRISPR) brings these tools closer to use in HD clinical trials
The news has recently been full of stories about CRISPR, a new "jaw-dropping" DNA-editing technology. Hype or hope?
Success in Huntington’s disease cells and mice for zinc finger drugs that reduce production of the harmful protein
Scientists combine gene therapy and stem cells to correct a genetic liver disease. Could this combo be retooled for H
Avoid the hype: HDBuzz presents ten 'golden rules' for reading a news story or press release about Huntington's disea
Scientists make precise changes to the DNA of a live animal. Could it work for human genetic diseases?