Columns

A survey of 1,700 Huntington’s disease patients that questioned issues such as symptoms, treatment, and disease management, revealed some surprising facts that can impact how physicians and researchers view and treat the condition. Particularly, the survey revealed that patients often lack support during the diagnostic process. GeneFo is an…

A recent review summarizes the scientific literature on behavioral and psychiatric symptoms of Huntington’s disease, noting that plenty of research is still needed to better understand and treat such symptoms, which traditionally have taken second stage to the disease’s movement problems. The review, “Changes in mental state…

Researchers have developed a new gene-editing tool that may improve the precision of future gene therapy efforts in Huntington’s disease, as well as numerous other genetic conditions. The study, “A chemical-inducible CRISPR–Cas9 system for rapid control of genome editing,” published in the journal Nature Chemical Biology,…

Teva Pharmaceutical presented encouraging top-line data from PRIDE-HD, a Phase 2 clinical trial evaluating the impact of pridopidine, an oral medication, on motor impairment in patients with Huntington’s disease (HD). Teva is now preparing for a larger clinical study in Huntington’s. The presentation took place at the 9th European…

Mice models of Huntington’s disease, infected with a parasite commonly found in humans, react in a different way to the infection than normal mice, researchers have revealed. The study suggests that infection and immune activation can impact the course of the neurodegenerative disease. Although researchers behind the study declined to speculate…

Attempts to block the activity of the mutant huntingtin gene, which causes Huntington’s disease, may have taken a big step forward. Scientists report they successfully silenced the gene for six months after a single injection in a mouse model of the disease. The study, “Deimmunization for gene therapy:…

Voyager Therapeutics has partnered with the CHDI Foundation to advance its VY-HTT01 gene-silencing therapy for Huntington’s disease (HD). The research alliance with CHDI builds upon an earlier partnership between CHDI and Sanofi Genzyme and includes financial support from CHDI to help in the preparation and filing of an FDA…

Using a new Huntington’s disease model featuring two types of disease affected neurons grown together in the lab, researchers showed that introducing a specific protein part reduced mutant huntingtin, as well as other molecular effects linked to the disease. Findings presented in the study titled “TRiC subunits enhance…

Inflammatory processes in both the brain and the rest of the body are likely an integral part of Huntington’s disease, according to a review of published data on the topic. The report, “Neuroimmunology of Huntington’s Disease: Revisiting Evidence from Human Studies,” published in the journal Mediators…

Gene therapy using mesenchymal stem cells for Huntington’s disease is showing promise in mouse studies, and preparations are underway to possibly move it into clinical testing. Before the technique might be ready for human trials, however, scientists need to master a few more steps, using larger animal models to investigate the therapy’s…