A recent paper links CAG repeat expansion with widespread RNA changes, especially in brain cells vulnerable to HD. “Knocking out” certain DNA repair genes that go awry in HD can have positive effects on features of HD in mice.
Scientists have found more people have the genetic changes that underlie repeat expansion diseases, like HD, than previously thought. This new data tells doctors to consider expansion diseases more when diagnosing people with neurological symptoms.
Making babies: HDBuzz's feature article - updated for 2024 - on fertility technologies that can help at-risk people to have HD-free children
Scientists have looked at CAG expansions in brains from people with HD to see which cells are affected
A new study from researchers at Thomas Jefferson University delves into the details of how genetic modifiers of Huntington’s disease work.
Voyager Therapeutics is shifting towards a new technology to deliver gene therapy and away from a planned HD clinical trial. But this could lead to less invasive drugs in the long run, and many other companies are working on HD gene therapies.
HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs
HDBuzz reports from the annual Huntington’s disease therapeutics conference in Palm Springs
Scientists screen the ENTIRE genome to find new potential therapeutic targets for HD. This ambitious study provides a wealth of data for HD researchers