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?
Data from GENERATION HD1, the Phase 3 clinical trial testing the huntingtin-lowering drug tominersen, have just been published in a scientific journal. The trial ended a while back, so why is this an important milestone, and what’s next?
Many diseases are caused by repetitive DNA sequences. Understanding the regulation of those repetitive sequences may hold the key for unlocking therapeutics for Huntington’s disease. A team from Toronto has just advanced our understanding.
The HD pipeline is rich and varied. Let's talk about some out-of-the-box approaches for developing drugs for HD that don't involve huntingtin lowering.
