People genetically susceptible to Huntington's disease often see their movement, mood, and cognition decline slowly over time ...
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 ...
PTC518 is designed to reduce levels of the mutated huntingtin protein that leads to ... Novartis also licensed rights to a pair of potential gene therapies for Huntington's and SMA from Voyager ...
The balance of protein health within cells, a process known as proteostasis, is critical for maintaining cellular ...
The authors develop and validate mFISHseq, a spatially informed assay that tackles several unmet needs in breast cancer, including discordance in molecular subtyping and prognostic risk and ...
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 ...
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 ...
Jan. 6, 2025 — A new study has identified 11 genes that may hold the key to understanding the brain's response to these pervasive chemicals commonly found in everyday ...
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 ...
Impact and progress Frontiers' impact Progress Report 2022 All progress reports ...