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:…
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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…
A multi-institutional study led by a team of biomedical researchers in the lab of Hui-Chen Lu at Indiana University at Bloomington, Indiana, has discovered evidence indicating that the enzyme nicotinamide mononucleotide adenylyl transferase 2 (NMNAT2) may have protective qualities against debilitating effects of certain degenerative brain diseases called proteinopathies, that…
Olesoxime, a drug shown to have neuroprotective properties, may have beneficial effects in Huntington’s patients through reduction of calpain-mediated huntingtin cleavage, according to the study “The calpain-suppressing effects of olesoxime in Huntington’s disease,” published in the journal Rare Diseases. Olesoxime is a small molecule…
The Weill Family Foundation and Joan and Sanford I. “Sandy” Weill have donated $185,000,000 to the University of California, San Francisco, to establish the UCSF Weill Institute for Neurosciences in an effort to expedite the development of new therapies for brain and nervous system diseases, including Huntington’s disease and Alzheimer’s…
A research report discusses the use of six versions of a new type of molecule, known as xyloketal B, for the treatment of Huntington’s disease. Scientists tested the six possible drugs in a worm model of Huntington’s disease, Caenorhabditis elegans. Results of the study,  “Xyloketal-derived small molecules show protective effect…
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Researchers Decrease Accumulation of Damaging Huntington’s Disease Protein Using New Method in Mice
A recent study published in the journal Neuron by University of California at Irvine researchers revealed that the protein PIAS1 regulates the accumulation of a mutant protein in Huntington’s disease, and its expression associates with disease pathogenesis in a mouse model of the disease. The study, “PIAS1 Regulates Mutant Huntingtin Accumulation…
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