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:…
News
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…
Vaccinex Inc. a clinical-stage biotechnology company recently announced that their drug candidate, VX15 developed for the treatment of Huntington’s disease (HD) received orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA).
A new study from researchers in Spain may increase understanding of a less recognized aspect of Huntington’s disease (HD): cognitive problems. The article examines the possible causes of cognitive difficulties in HD and how they might be addressed through new therapies. The report, “Cognitive Dysfunction in Huntington’s Disease: Mechanisms and…
In a recent study, the modification of a specific component of the huntingtin protein reduced neurodegenerative symptoms in an animal model’s brain and offered hope for future therapies that could effectively treat Huntington’s disease in humans. The study, “Serine 421 regulates mutant huntingtin toxicity and clearance in mice,” was published in the…
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