Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Recent days have seen a slew of news emerging regarding the use of something called genome editing as a potential therapy for genetic diseases like Huntington's Disease. These approaches, which include exotic sounding tools like zinc finger nucleases and CRISPR/Cas9, differ from more traditional ways reducing the impact of the HD mutation on cells. What's new in this exciting area of research?
A recent article in the UK newspaper the Daily Telegraph has HD families very excited. The title, "First drug to reverse Huntington’s disease begins human trials", certainly sounds exciting! But what's really going on? HDBuzz is here to help us untangle hope from hype in the huntingtin lowering world.
Today brings news that the first Huntington's Disease patients have been successfully dosed with gene silencing drugs targeting the HD gene. These brave volunteers are the first HD patients to ever be treated with drugs designed to attack HD at its root cause, a treatment approach with huge potential. What about this news has us so excited?
When patients participate in clinical trials, there needs to be some type of readout to determine whether the new treatment worked. It’s important to know two key things: What to measure and how to measure it. In the case of HD, these obstacles have vexed scientists and doctors for years. The latest research comes up with a clever new approach to overcome both challenges in a new way. These results could offer a valuable tool to study new HD therapeutics entering clinical trials.
A new clinical trial just announced for 2015 aims to test a “huntingtin lowering” therapy, called an antisense oligonucleotide (ASO), that attacks mutant huntingtin directly. We’re extremely excited—it’s the first-ever human HD trial to fight HD at the root of the problem, and has shown great promise in animal models. What’s the scoop?
Pharmaceutical giant Roche recently described a new drug delivery technology they call the 'brain shuttle'. Why has Huntington's disease been mentioned in so many press releases about this technology, and how much can we hope to gain from this new advance?
The double-helix of our DNA contains a backup copy of every gene. Sometimes the backup DNA produces hidden 'messages' that can alter things in our cells. Researchers have now discovered a message in the backup copy of the Huntington's disease gene, which appears to be able to influence how much huntingtin protein is made.
