Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
Some techniques aimed at lowering mutant huntingtin can also affect the normal form of the protein. With clinical trials underway, it’s all the more important to understand the role of normal huntingtin in the adult brain. Researchers recently inactivated the huntingtin gene in healthy adult mice of different ages. They found that this could cause neurological and behavioral problems. Mice aren’t perfect for modelling human brains, and no huntingtin-lowering drug would remove the protein completely - but this research supports the need for continued caution as we test drugs that lower normal huntingtin.
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?
Recent days have seen a torrent of news stories about a new technology, called CRISPR, which has been described as having potential application in Huntington's disease. Is this new technique as cool as it sounds? Possibly — but, as always, the truth is more complicated than the headlines suggest.
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?
Gene therapy could correct spelling mistakes in our DNA, while stem cells promise transplant operations that don't need powerful immune suppressing drugs. Scientists have now combined the two techniques in a genetic liver disease. Several challenges remain before this could work in Huntington's disease though.
Real progress is being made on the road to Huntington's disease treatments, but sometimes it feels like scientists promise more than they can deliver. So, HDBuzz has come up with ten 'golden rules' to help you decide whether a news story or press release offers genuine promise for HD, or whether its claims should be taken with a pinch of salt.
What if we could edit the DNA of patients to remove the Huntington's disease mutation altogether? Sounds like science fiction, but new research in an animal model of hemophilia suggests that it can work - and now HD researchers are on the case.