News

GeneFo will offer a free webinar on how adding humor to Huntington’s patients’ lives can help improve their symptom management, emotional wellbeing, ability to cope with adversity, and social and work interaction. The webinar, which is aimed at caregivers as well patients, will start at 1 p.m. U.S. Eastern…

Three entities — the Clinical Data Interchange Standards Consortium (CDISC), the Critical Path Institute (C-Path) and the CHDI Foundation (CHDI) — have announced that the Huntington’s disease (HD) Therapeutic Area User Guide (TAUG-HD v1.0) is up for public review through Nov. 10. TAUG-HD v1.0 was developed through the Coalition…

Huntington’s disease causes hearing problems, a study with a small sample size suggests. The research, “Auditory dysfunction in patients with Huntington’s disease,“ was published in Clinical Neurophysiology. A severe, inherited neurodegenerative disease, Huntington’s is characterized by movement problems, cognitive deterioration and behavioral disorders. Many people with other neurodegenerative…

Researchers generated the first human line of Huntington’s disease induced pluripotent stem cells (iPSCs) that do not express the dysfunctional protein underlying the condition. The cells represent a potential resource to investigate the function of the huntingtin protein in vitro (in a lab culture dish or test tube). The study,…

AC Immune has identified antibodies that target the alpha-synuclein and TDP-43 proteins associated with neurodegenerative diseases such as Huntington’s. The discovery could be used to develop treatments for such diseases, the company said. Antibodies are immune system cells that can recognize and generate a response to invaders. “Many neurodegenerative diseases share…

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…