New work from researchers in London uses mice to narrow in on the number of CAG repeats needed to cause symptoms of Huntington’s disease. Their work points to fewer than 185 CAGs as a threshold.
Is someone cutting onions? Expanded huntingtin can now be detected in tears to help scientists track disease progression.
Scientists in Massachusetts have recently advanced our understanding of how repetitive sequences in DNA can disrupt the creation and editing of genetic messenger molecules in cells, and how this could lead to the production of harmful proteins.
Scientists have looked at CAG expansions in brains from people with HD to see which cells are affected
Check out research updates from Day 1 of the 2024 HD Therapeutics Conference #HDTC2024
Casgevy is the first CRISPR-based drug to make its way through the approval process, all but curing Sickle Cell Disease and it’s paving the way for similar drugs targeting other diseases. Is Huntington’s disease next?
uniQure ushered in the end of the year by releasing some promising data from their huntingtin-lowering gene therapy trials
