Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Isis Pharmaceuticals and Roche have announced a multi-million dollar deal to support the development of 'gene silencing' drugs to human trials. This is big news that secures the future of these exciting drugs for Huntington's disease.
Everyone has two copies of the huntingtin gene but Huntington's disease is caused by a copy that's extra-long. New research shows that cells have different controls for how the normal and extra-long instructions are used to make protein. These controls on the protein-making process may be targets for developing drugs for HD.
Designing drugs that tell our cells to make less of the harmful mutant huntingtin protein is one of the most promising approaches to treating Huntington's disease. Most huntingtin-lowering attempts so far have tried to 'shoot the messenger' rather than attacking the source of the message - the DNA itself. Now, two independent reports of success in HD mice have given a boost to 'zinc finger' drugs - which interact directly with the HD gene itself. It's early days for this new technology: what do we know, and what challenges are ahead?
The Hereditary Disease Foundation, or HDF, is a key player in the world of Huntington's disease research. At the HDF's recent biennial scientific meeting in Cambridge, Massachusetts - 'The Milton Wexler Celebration of Life and Creativity' - HDBuzz met Nancy and Alice Wexler, the remarkable sisters at the heart of the HDF's work.
Watch the video of Ed Wild's address to the European Huntington's Association annual meeting in September 2012, summarizing how therapies research works and explaining the most exciting ways researchers are trying to study and treat HD.
After huge leaps forward in recent years, we're edging ever closer to human trials of huntingtin lowering or 'gene silencing' as a potential treatment for Huntington’s Disease. Newer, better and safer techniques are always welcome and the announcement of ‘single-stranded RNA’ silencing is causing quite a hubbub. What’s it all about?
Jeff Carroll and Ed Wild present EuroBuzz episode 2 - bringing the European HD Network Meeting direct to you, in plain language. Watch online!
Our second daily report from the European Huntington’s Disease Network 2012 Meeting in Stockholm. Video of both EuroBuzz evening sessions will be available to watch on HDBuzz.net next week.
Drugs called anti-sense oligonucleotides, or ASOs, are one way of silencing the gene that causes Huntington's disease. A new publication in the journal Neuron suggests that ASO gene silencing reaches further in the brain than other methods, lasts longer and is safe.