The molecule used in the lab tests at the U of A was produced both naturally and synthetically through chemical production. This same molecule has been used in clinical trials for the treatment of Parkinson's and other neurodegenerative diseases, so using this molecule to treat patients with Hun...
However, even though the quantification of blood-based mHTT is a reliable technically, it is important for a biochemical biomarker to predict the clinical onset of symptoms or reflect the progression of clinical manifestations. Weiss et al. first observed the association between blood-based mHTT le...
To that end, researchers at Children’s Hospital of Philadelphia (CHOP) have created a “dimmer switch” system that employs Novartis’ investigational Huntington’s disease medicine branaplam (LMI070) as a regulator to fine-tune the quantity of proteins generated from a gene therapy. The investiga...
Edinburgh (Molecular Medicine Centre, Western General Hospital, Department of Clinical Genetics): Maureen Edwards; Teresa Hughes (Scottish Huntington's Association); Marie McGill; Pauline Pearson; Mary Porteous; Paul Smith (Scottish Huntington's Association); Adam Zeman. Exeter (Heavitree Hospital): ...
In the present study, we investigated the presence and nature of changes in the fornix in the limbic system in HD using three sets of clinical material. We assessed the volume of the fornix using MRI from a 3 T scanner in the IMAGE-HD cohort study, a multimodal neuroimaging study including...
Yuwei Yao1, Yuyin Pan1, Xue Wen1, Lixiang Ma2, Saiyin Hexige1, Yu Ding1, Shouqing Luo3, Boxun Lu1 1State Key Laboratory of Medical Neurobiology, Huashan Hospital, School of Life Sciences, Collaborative Innovation Center for Genetics and Development, Fudan University, Shanghai 200438, China...
Participants. Twelve pre-symptomatic carriers (preHD) of the HD-gene mutation and 22 age and sex matched healthy controls were recruited from the Huntington's disease clinic at Ulm University Hospital between 2012 and 2014. Far from onset preHD participants were eligible when they had a ...
All AAVs were made by the Research Vector Core at The Children’s Hospital of Philadelphia. Titers (measured in viral genomes/mL) were determined by qPCR. Cardiac Stress Induction Isoprenaline (ISO, Sigma) was dissolved in sterile PBS and administered by continuous infusion using implantable mini...
F.M. Kirby Neurobiology Center, Boston Children's Hospital, Harvard Medical School, Boston, Massachusetts, USA Daniel K Wilton & Beth Stevens Department of Neurobiology and Behavior, University of California, Irvine, Irvine, California, USA., Alvin R King, Delaram Sharifabad, Sara T Winokur &...
We would like to thank the patients and control subjects who donated samples and took part in these studies, as well as the Track-HD investigators, particularly Professors Blair Leavitt (UBC, Vancouver), Alexandra Durr (Pitié-Salpêtrière Hospital, Paris) and Raymund Roos (LUMC, Leiden). Some...