News

uniQure recently published results from preclinical studies of its gene therapy program, AMT-130, indicating that a one-time administration of AAV5-delivered therapy into the central nervous system can block the mutant HTT gene that causes Huntington’s disease (HD). These findings, in the article “Design, Characterization, and Lead Selection of Therapeutic miRNAs…

Two nonprofit groups advocating for those with Huntington’s disease (HD), HD Reach and the North Carolina Chapter of the Huntington’s Disease Society of America (HDSA-NC), have announced a second daylong educational event for people with HD, their families, and caregivers. This year’s NC HD “Hope for Health, Hope for Life” will be at held…

University of California at Davis researchers have, for the first time, successfully tested human stem cells (MSCs) as a delivery system for a key protein growth factor in mouse models of Huntington’s disease (HD), an important step for the development of novel therapies. The research article, “Human Mesenchymal Stem Cells…

The English Parliament recently listened to a young scientist, Emma Yhnell, 25, explain her research into the potential of computer games to “train the brain” of Huntington’s disease (HD) patients. The research associate from Cardiff University in Wales attended Parliament on March 7 to present her novel approach to a range of politicians and…

Researchers at Philadelphia’s Thomas Jefferson University investigated the physiological and pathological role of MICU1 (Mitochondrial Calcium Uptake 1), a protein that controls calcium by the mitochondria, a process involved in cell survival. Results suggest that strengthening this protein’s function and inhibiting mitochondrial calcium uptake could be a novel therapeutic approach…

New Huntington’s research sheds light on the link between the effects of the disease’s underlying mutation on brain structure and function, and consequent behavior anomalies. Studying the gene mutation’s expression in a precise brain area of songbirds, scientists observed how the mutation affects specific neurons and changes behavior, findings that could lead to the…

Emory University researchers deleted the Huntington’s disease-causing protein, huntingtin, in the brains of adult mice, and observed that such deletion was safe in adults though not in relatively newborn mice. The study, “Ablation of huntingtin in adult neurons is nondeleterious but its depletion in young mice causes acute pancreatitis”…

The Huntington Disease Care, Education and Research Center at Georgetown (HDCERC) was recently designated a HDSA Center of Excellence for 2016, a recognition given by the Huntington’s Disease Society of America to facilities taking a multidisciplinary approach to the treatment of people with Huntington’s disease, and excelling in the care given patients, the support shown to their…

Huntington’s disease (HD) patients are known to have a dysregulation of cholesterol metabolism. Now, researchers have shown that manipulation of cholesterol levels in striatal neurons can improve motor deficits in an animal model of Huntington’s. The study, “CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s…

A Huntington’s researcher at the University of Toronto — Rachel Harding — will be the first biomedical scientist known to share documentation of her experiments in real-time. She is also inviting the public to scrutinize her work through her blog, another move that she hopes will accelerate research into Huntington’s disease. “This…