A Genetic Lottery - a Column by Becky Field

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…

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…

Cambridge, England-based light therapy specialist Lumie will supply 24 of its most powerful light boxes, called Lumie Brazil, for use in a Huntington’s disease research study at the Cambridge School of Clinical Medicine’s Neurology Unit. The Lumies are being donated to…

Huntington’s disease (HD) patients are known to have a dysregulation of cholesterol metabolism. Now, researchers have shown that manipulation of cholesterol levels in striatal neurons can improve motor deficits in an animal model of Huntington’s. The study, “CYP46A1, the rate-limiting enzyme for cholesterol degradation, is neuroprotective in Huntington’s…

A Huntington’s researcher at the University of Toronto — Rachel Harding — will be the first biomedical scientist known to share documentation of her experiments in real-time. She is also inviting the public to scrutinize her work through her blog, another move that she hopes will accelerate research into Huntington’s disease. “This…

Researchers from the École Polytechnique Fédérale de Lausanne (EPFL), Switzerland, led by Dr. Matteo Cornaglia, PhD, have developed a new experimental device that can assist scientists in the analysis of molecular pathway studies related to complex human dysfunctions, such as neurodegenerative diseases like Huntington’s (HD). The device is intended for use with the…