Huntington’s disease research news. In plain language. Written by scientists. For the global HD community.
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?
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.