Inherited repeat mutations in the HTT gene expand in neurons over decades ... neurons might not be inherently more affected by mutant huntingtin than other cell types are, but instead could ...

The disease is caused by a mutation in the gene coding for huntingtin, which causes the formation of an abnormally long and unstable form of the protein that is chopped up by cellular repair ...

Dec. 10, 2024 — In a single IV injection, a gene therapy targeting cBIN1 can reverse the effects of heart failure and restore heart function in a large animal model. The therapy increases the ...

It seeks to understand the process of trait inheritance from parents to offspring, including the molecular structure and function of genes, gene behaviour in the context of a cell or organism (e.g ...

Huntington's disease is a rare genetic, autosomal dominant, neurodegenerative disease, caused by CAG repeat expansions in the HTT gene, that is polymorphic on the healthy allele and contains more than ...

“We are excited to present on our Huntingtin-lowering clinical trials at the HDYO International Congress this month,” said Sergey Paushkin, Chief Scientific Officer and Head of R&D at Skyhawk, “with ...

Mar. 14, 2025 — Researchers have discovered that cancer cells suppress 'poison exons' -- genetic elements that act as an off switch for protein production -- in a key gene called TRA2 ...

Leora Fox trained as a Huntington's Disease scientist, and obtained her PhD in 2016 at Columbia University, where she studied HD mouse models and how brain cells dispose of huntingtin protein. After ...

Ed is a neurologist at the National Hospital for Neurology and Neurosurgery, London, UK, and a Professor of Neurology at University College London where he is Associate Director of the Huntington’s ...