Novel RNAi therapy silences mutated Huntington’s disease gene and reduces symptoms

PUBLIC RELEASE DATE:

21-May-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, May 21, 2014A targeted gene silencing strategy blocks production of the dysfunctional huntingtin (Htt) protein, the cause of Huntington's disease, a fatal, inherited neurodegenerative disorder. The effectiveness of this RNA interference (RNAi) approach in reducing levels of mutant Htt protein and disease symptoms in a mouse model of the disease is described in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website.

Lisa Stanek and coauthors from Genzyme (Framingham, MA) used an adeno-associated viral (AAV) vector to deliver a targeted nucleic acid sequence called a small interfering RNA (siRNA) into the cells of affected mice. The siRNA selectively binds to the mutated gene, blocking disease-causing Htt production. The authors present data demonstrating the ability to deliver the therapeutic RNAi into the cells, reduce mutant Htt levels, and impact behavioral deficits in the mice without causing any noticeable neurotoxicity, in their article "Silencing Mutant Huntingtin by Adeno-Associated Virus-Mediated RNA Interference Ameliorates Disease Manifestations in the YAC128 Mouse Model of Huntington's Disease."

"The Genzyme group uses state-of-the-art delivery technology and a gene silencing approach to generate very promising preclinical data for Huntington's disease," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

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Novel RNAi therapy silences mutated Huntington's disease gene and reduces symptoms

University of Utah names dean for College of Social and Behavioral Science

The University of Utah's College of Social and Behavioral Science has named Cynthia Berg its new dean and Richard Forster its new associate dean.

Jordan Allred,

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SALT LAKE CITY The University of Utah's College of Social and Behavioral Science has named a new dean and associate dean.

Cynthia A. Berg, professor of psychology at the U., has been named the dean of the college effective July 1, pending approval by the board of trustees. Richard R. Forster has been appointed associate dean

Berg has served as interim dean of the college since 2013 and was chairwoman of the psychology department from 2008 to 2011. She joined the U. faculty in 1987 and holds a doctorate in psychology from Yale University.

Berg's research involves developmental and health psychology, examining how individuals in close relationships deal with stress, decision-making, and problem-solving around chronic health conditions such as diabetes and cancer. She has been published and funded by the National Institutes of Health.

Forster has been interim associate dean since 2013. He is a professor of geography and joined the U. faculty in 1999. His research focuses on using remote sensing data to derive information about snow and ice conditions at Earth's surface specifically how glaciers and seasonal snow cover response to climate change.

Forster holds a doctorate in earth and atmospheric sciences from Cornell University.

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University of Utah names dean for College of Social and Behavioral Science

Is Social Science Research in the National Interest?

A battle is raging in the House of Representatives over an effort to clip the National Science Foundations wings

Today the science committee of the U.S. House of Representatives will begin debate on a bill key to national research funding and priorities. The Frontiers in Innovation, Research, Science and Technology (FIRST) Act of 2014 (H.R. 4186) would, among other things, require the National Science Foundation to cut research for social sciences and economics and certify to Congress that each taxpayer-funded grant it issues is in the national interest.

Last week Scientific American published an interview with Neal Lane, a former White House science advisor, that was critical of the FIRST Act. Rep. Lamar Smith (RTexas), chair of the House Science, Space and Technology Committee and co-sponsor of the FIRST Act, contacted us and asked for a chance to respond. We publish Rep. Smiths essay below along with a commentary on the FIRST Act by Hunter Rawlings, president of the Association of American Universities, and Peter McPherson, president of the Association of Public and Land-grant Universities.The Editors

The Role of Congress Is to Set Priorities for Research By Rep. Lamar Smith (RTexas)

Technological advancement drives U.S. economic growth. Sustained public and private sector investments in mathematics, engineering, computer science and biology have led to new and expanded industries. Advancements in these fields have created millions of jobs that have supported generations of American families. Since World War II the U.S. has led the world in research and development. And America still spends more on R&D than any other nation.

But experts warn that other nations are catching up. Recent forecasts suggest that China will overtake the U.S. in total R&D spending by 2022. China can already lay claim to the worlds fastest supercomputer, an area of long-standing U.S. dominance. And the World Bank reports that Chinas high-tech exports are more than double those of the U.S.

Unfortunately, there has been a shift in priorities at the National Science Foundation (NSF) away from basic research in engineering and the physical sciences toward social/behavioral/economic (SBE) studies. In his budget proposal for fiscal year 2015 the president proposes to increase SBE by more than 5 percent while freezing or cutting funds for engineering and physical sciences.

I believe the Frontiers in Innovation, Research, Science and Technology (FIRST) Act sets a better course for taxpayer-funded research. The FIRST Act refocuses taxpayer investments on basic research in engineering, mathematics, computer science and biology, increasing funding for those NSF directorates by between 7 and 8 percent for the next fiscal year. These are the areas singled out by The National Academies of Sciences, Engineering and Medicine as the primary drivers of our economic future. These are the areas of science with the greatest potential to yield transformational new technologies, catalyze new industries and businesses as well as create millions of new jobs.

Setting priorities for federally funded research is not new. During the 1970s, 1980s and 1990s, under both Republican and Democrat administrations, Congress regularly defined annual appropriations and authorizations by academic field. Neal Lane, former science advisor to Pres. Bill Clinton who criticized the FIRST Act in a recent Scientific American interview, apparently has forgotten that Clinton signed the NSF Authorization Act of 1998, which was just as specific in delineating research priorities as the FIRST Act.

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Is Social Science Research in the National Interest?

IIT Center For Nanotechnology Innovation@NEST, ITALY (MIT-LS 2014) – Video


IIT Center For Nanotechnology Innovation@NEST, ITALY (MIT-LS 2014)
IIT Center for Nanotechnology Innovation@NEST MIT-LS 2014 Pitch Presentation - March 31, 2014 Center for Nanotechnology Innovation@NEST is a center of Istituto Italiano di Tecnologia embedded...

By: Meet in Italy for Life Siences

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IIT Center For Nanotechnology Innovation@NEST, ITALY (MIT-LS 2014) - Video