Researchers got surprisingly lucky when looking for drug molecules to pull mutant huntingtin protein into a cellular garbage disposal machine
Exciting new Huntingtin Lowering work from @SangamoTx and @CHDIfoundation using "Zinc Fingers" to shut down expression of the mutant Huntingtin gene. More details on this exciting new technique here.
Evolving CRISPR-Cas9 techniques can now be used to edit the HD gene in a living mouse brain.
Two recent studies show how a cellular border control system goes wrong in HD, opening new avenues for HD research.
Is HD a developmental disease? HD stops neurons migrating in developing brain, but maybe we can get them going again
A surprising new mouse study suggests the mutant HD gene may do some of its damage during embryonic development
Harmful misfolded huntingtin can travel between brain cells via messenger particles called exosomes
An existing drug could give a boost to HD brain cells – but it may need more testing before it’s back in the clinic
Fingolimod, a drug used in multiple sclerosis, prevents memory problems in HD mice. Would it work in patients?