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…
Researchers at the University of Auckland in New Zealand showed that the pattern of neuronal loss in three cortical brain areas in Huntington’s disease corresponds to the predominant symptom type in a patient. The study, “Symptom heterogeneity in Huntington’s disease correlates with neuronal degeneration in the…
A new study by researchers at the Salk Institute, in California, sheds light on how a major movement center of the brain called the striatum might work. The striatum is involved in several nervous system diseases including Huntington’s disease where neurons in the region progressively degenerate, leading to severe motor and cognitive problems and…
Despite efforts to develop therapies to treat Huntington’s disease (HD), so far no treatment has been shown to effectively stop, slow, or reverse disease progression. Now, an animal study suggests that treating mice with the experimental drug laquinimod can improve some neuropathologic and behavioral features of Huntington’s. The study, titled “Laquinimod…
Get regular updates to your inbox.