News

A new version of the CRISPR-Cas9 gene editing technique designed to target RNA molecules can fix the underlying cause of Huntington’s disease and similar disorders, say researchers at the University of California San Diego. Their study, “Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9,” appeared in…

Researchers in England and Wales have identified a gene linked to disease progression in Huntington’s disease, shedding new light on the poorly understood mechanisms that cause the disorder. Although the results — published in the journal The Lancet Neurology —hold no immediate prospect of leading to a cure, researchers say…

Aggregates of mutant huntingtin protein have been associated with cellular toxicity in Huntington’s disease. Now researchers have discovered that isolated forms of mutated huntingtin are actually responsible for major molecular changes in cells that will drive toxicity. The study, “Transcriptional profiles for distinct aggregation states of mutant Huntingtin exon…

Dutch pharmaceutical company uniQure says it plans to conduct preclinical studies leading to clinical testing of a gene therapy in patients with Huntington’s disease. The company is developing AMT-130, an experimental therapy that aims to block production of the mutated and aggregation-prone huntingtin protein in patients. Composed of a small…

Researchers used gene editing to suppress the activity of protein mutations in the brains of mice with  Huntington’s disease. Suppressing the huntingtin protein alleviated early signs of the disease and reduced the mice’s movement problems, the study reported. The research, “CRISPR/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of…

Therapies aimed at reducing the levels of a protein called HDAC3 may improve cognitive decline in Huntington’s disease, according to the results of a new study. The study titled, “A selective inhibitor of histone deacetylase 3 prevents cognitive deficits and suppresses…

A snapshot of a key brain receptor in action might advance the understanding of disease processes in Huntington’s disease and allow scientists to identify new treatments, researchers at Columbia University Medical Center (CUMC) hope. Their work, published in the journal Nature, describes how the neurotransmitter glutamate binds to…

Researchers have created the first freely available data network — called HDNetDB — that aims to advance research into Huntington’s disease and speed up drug discovery for the incurable condition. The approach, described in an article in the journal Scientific Reports, particularly focuses on the complex molecular workings…

Wave Life Sciences will start two Phase 1b/2a clinical trials to investigate the safety and tolerability of its products WVE-120101 and WVE-120102 in Huntington’s disease patients. The trials will also look at the therapies’ effectiveness. “Wave’s PRECISION-HD program is the first to target the underlying cause of Huntington’s disease…

Huntington’s patients who are taking Xenazine for the twitching condition known as chorea can switch without concern to the newly approved Austedo, according to Austedo’s maker, Teva Pharmaceutical Industries. A Phase 3 clinical trial showed that the switch was safe and that Austedo (deutetrabenazine) did a better job of controlling chorea than…