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New study shows long-term safety of gene therapy in Parkinson's disease

July 27, 2016

New Rochelle, NY, July 27, 2016--New safety data from a study of patients with advanced Parkinson's disease five years after gene transfer-mediated delivery of the neuroprotective factor neurturin directly to patients' brains reveal no serious adverse events related to the treatment. The encouraging long-term safety profile of the surgically administered adeno-associated virus (AAV2)-neurturin gene therapy is described in an 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 until August 27, 2016.

In the article "Long-Term Safety of Patients with Parkinson's Disease Receiving rAAV2-Neurturin (CERE-120) Gene Transfer" William Marks, Jr., University of California, San Francisco, Tiffany Baumann, Isis Pharmaceuticals (Carlsbad, CA), and Raymand Bartus, RTBioconsultants (San Diego, CA), representing the CERE-120 Study Group, describe the study design, which greatly extended the length of follow-up of the 53 patients evaluated compared to previous studies for the purpose of identifying any safety issues that might present months or years after the treatment.

"The longevity of rAAV vector expression makes it particularly useful for treatment of chronic neurodegenerative 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. "These long-term data lend support to the evidence that rAAV will prove to be an important platform for treating this highly debilitating disease."
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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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