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Promising results with new gene therapy approach for treating inherited neurodegenerative diseases

July 28, 2016

New Rochelle, NY, July 28, 2016-- A new gene therapy approach designed to replace the enzyme that is deficient in patients with the inherited neurodegenerative disorders Tay-Sachs and Sandhoff diseases successfully delivered the therapeutic gene to the brains of treated mice, restored enzyme function, and extended survival by about 2.5-fold. The implications of these promising results for developing similar gene therapies for use in hu-mans and for targeting additional brain disorders are discussed in two articles published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The articles are part of a special issue on CNS disorders and are available free for download on the Human Gene Therapy website until August 28, 2016.

Both studies demonstrate the feasibility and efficacy of gene transfer in preclinical models. The articles are entitled "Novel Vector Design and Hexosaminidase Varieant Enabling Self-Complementary Adeno-Associated Virus for the Treatment of Tay-Sachs Disease", by Karumuthil-Melethil, et al.; and "Systemic Gene Transfer of a Hexosaminidase Variant Using a scAAV9.47 Vector Corrects GM2 Gangliosidosis in Sandhoff Mice", by Osmon et al.

Steven Gray, University of North Carolina at Chapel Hill, and Jagdeep Walia, Queen's University (Kingston, Canada), led a team of researchers from SickKids and University of Toronto (Canada), New Hope Research Foundation (North Oaks, MN), and University of Mannitoba (Winnipeg, Canada), in the successful development of a specialized adeno-associated virus (AAV) vector designed to deliver a gene coding for portions of the alpha and beta subunits of the enzyme that are defective in the Tay-Sachs and Sandhoff mice, respectively. The novel gene transfer vector, administered intravenously, was able to deliver the therapeutic gene to the brain and spinal cord, the targeted site of action.

"This important proof-of-concept study sheds important information on the optimal design of rAAV vectors for this class of disorders," says Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, Worcester, MA.
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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. Led by Editor-in-Chief Terence R. Flotte, MD, Celia and Isaac Haidak Professor of Medical Education and Dean, Provost, and Executive Deputy Chancellor, University of Massachusetts Medical School, 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 companion 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 contents for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Nucleic Acid Therapeutics, Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Mary Ann Liebert, Inc./Genetic Engineering News

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