A Genetic Lottery - a Column by Becky Field

Huntington’s disease is caused by excessive repeats of a portion of DNA, called CAG triplets, within the gene that codes for huntingtin (HTT). New research shows how the number of CAG repeats determines the final form and the aggregation profile of the resulting protein. The study, “The Aggregation Free Energy…

Gene therapy biotech uniQure is prioritizing its drug pipeline to develop treatments for Huntington’s disease, hemophilia B, and its partnered gene therapy programs associated with uniQure’s collaboration with Bristol-Myers Squibb (BMS) in cardiovascular disease. The company will restructure its research and development organization in the Netherlands and consolidate manufacturing in…

Researchers identified a new molecular mechanism related to mutant huntingtin (HTT) — messenger RNA — that may play a role in the development of Huntington’s disease. This finding may help define new therapeutic approaches to tackle the disease. The study, “Targeting CAG Repeat RNAs Reduces Huntington’s Disease Phenotype Independently Of…

Researchers identified several new biomarkers in mice that may help in the early diagnosis of Huntington’s disease in humans, according to a new study. These biomarkers may also be useful to evaluate patients’ responses to potential treatments. The Stanford University School of Medicine study, “Potential Biomarkers To Follow The…

A drug in clinical development by Takeda for schizophrenia, called TAK-063, was seen to reduce neuron death and improve symptoms in mice with Huntington’s disease. But the data needs to be confirmed in other animal models of Huntington’s, and it is too early to say if the drug would also benefit…

Critical Path Institute (C-Path) and CHDI Foundation have joined forces to create a consortium geared toward clearly defining regulatory pathways for faster approval of new Huntington’s disease (HD) treatments. The new HD Regulatory Science Consortium (HD-RSC) will gather participants from the Huntington’s disease community to expedite regulatory endorsement and to facilitate…

By using computational modeling, researchers have demonstrated that at the early stages of mutant huntingtin (mHtt) aggregation, a molecular feedback loop works to prevent the effects of the toxic aggregates, until a tipping point is reached. Because the study, “Systemic study of a natural feedback loop in…

Researchers at the University of Cambridge, United Kingdom, recently investigated the effect of methamphetamine on the circadian clock of a genetically engineered mouse model of Huntington’s disease (HD) and found that the disruption caused behavioral abnormalities in a gene dose- and age-dependent manner. The study, “Progressive gene dose-dependent disruption…

An extensive research project has mapped out quality of life issues associated with Huntington’s disease, focusing on matters most important to patients and converting the identified issues into a new “smart” tool that enables patients to easily communicate their concerns with physicians. The project, funded by the National Institutes of…

Researchers at the University of Texas identified a potential drug that could be particularly effective in treating Huntington’s disease using a new platform and the microscopic worm Caenorhabditis elegans.