Clinical advances in gene therapy for central nervous system disorders


New Rochelle, NY, July 25, 2016–The encouraging results of early stage clinical studies and the tremendous amount of preclinical data demonstrating the feasibility and promise of gene therapy to treat disorders of the central nervous system (CNS) are driving new advances for the treatment of both genetic and acquired neurodegenerative diseases. Recent progress in therapeutic adeno-associated virus (AAV)-mediated gene transfer strategies and prospects for the future are presented in a Review article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is part of a special issue on CNS disorders and is available free for download on the Human Gene Therapy website.

In the article "Adeno-Associated Virus-Based Gene Therapy for CNS Diseases" coauthors Michael Hocquemiller, Laura Giersch, Mickael Audrain, Samantha Parker, and Nathalie Cartier, of Lysogene (Neuilly sur Seine), Université Paris Descartes (Paris), Université Paris-Sud and Université Paris-Saclay (Orsay), and CEA, DSV, I2BM, MIRCen (Fontenay-aux-Roses), France, describe the expanding scope of CNS diseases being targeted with gene therapy. These include lysosomal storage diseases, Alzheimer disease, Parkinson's disease, amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA). The researchers discuss the rapid advances in the development of new viral vectors and compare the advantages and limitations of different gene therapy delivery strategies to treat CNS dis-orders.

"The global disease burden of neurodegenerative diseases is increasing dramatically with the aging of populations in the developed world," 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. "Gene therapy promises to be a critically important part of the response of the biomedical research community to this enormous public health challenge."


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.

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