HD Insights: Volume 11

HD Insights: Volume 11

Meet the Attorney

April 1st, 2015 rtullio VITAL SIGNS NAME: Frank J. Sasinowski, MS, MPH, JD POSITION: Director, Hyman, Phelps & McNamara, PC; Member, Board of Directors, National Organization for Rare Disorders (NORD) EDUCATION: JD, Georgetown University Law Center; MS, Nutritional Sciences, University of California at Berkeley; MPH, University of California at Berkeley; BS, Biological Sciences and Genetics, Cornell University HOBBIES: Contemplation
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Research Round-Up

April 1st, 2015 rtullio By: Lise Munsie, PhD In stem cell research… Generating GABAergic medium-sized spiny neurons (MSNs) from human pluripotent stem cell (hPSC) sources is a rapidly evolving technique that holds much promise for HD modeling and treatment. Arber and colleagues describe a new method of differentiating hPSCs to MSNs using Activin A (activin).1 Activin is part of
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Editor’s Letter

April 1st, 2015 rtullio Welcome to the 11th edition of HD Insights. We are pleased to continue our mission to promote, disseminate, and facilitate research in Huntington disease. We are grateful to the Huntington Study Group (HSG), our sponsors, and our more than 2800 subscribers for their continuing support. This issue investigates potential therapeutic strategies for HD treatment and
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Update on HD Disease-Modifying Strategies on the Horizon

April 1st, 2015 rtullio By: Daniel Zielonka, MD, PhD More than 20 years after the identification of the wildtype HTT gene and the mutated allele (mHTT) that causes HD, we still have no cure, although there are a few therapeutic options for HD symptoms. The most promising disease-modifying strategies on the horizon focus on HTT lowering (Figure), and some
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Unraveling the Cause of Dysfunctional Autophagy in HD

April 1st, 2015 rtullio By: Dagmar Ehrnhoefer, PhD Mutant huntingtin (mHTT) is the direct cause of HD, and many exciting new treatment strategies are aimed at lowering its levels. The primary approach is to prevent generation of mHTT. However, there are also intracellular mechanisms to clear mHTT once it has been generated, and even mHTT aggregates can be degraded.
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Teva acquires Auspex for $3.5 billion

April 1st, 2015 rtullio By: Ryan E. Korn, BA On March 30, 2015, Auspex Pharmaceuticals, Inc. (La Jolla, CA, USA) and Teva Pharmaceutical Industries, Ltd. (Tel Aviv, Israel) announced that Teva will buy Auspex in a cash deal valued at $3.5 billion. The deal will enhance Teva’s central nervous system portfolio with the addition of Auspex’s deuterated compounds for
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Greasing the Huntington Disease Brain: Palmitoylation as a therapeutic target

April 1st, 2015 rtullio By: Shaun S. Sanders, PhD Much of our current understanding of the functions of wildtype HTT and of the pathological processes of mHTT comes from studies involving its interacting partners. Many studies seek to determine the full spectrum of HTT interacting proteins (HIPs) and to understand how their interactions are altered by mHTT, since interactions
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Highlights from the 10th Annual HD Therapeutics Conference

April 1st, 2015 rtullio By: Francesca Cicchetti, PhD This year’s 10th annual CHDI HD Therapeutics Conference, held February 23–26, 2015 in Palm Springs, California, brought together over a hundred researchers from academia and the private sector. The four-day meeting offered an exciting program covering a wide variety of topics related to HD research, from new methodologies to tackle fundamental
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