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What causes gene transfer to trigger T cell activation and exhaustion?

April 27, 2017

New Rochelle, NY, April 27, 2017--Researchers are beginning to gain a clearer understanding of how the immune system responds, in both a reactive and tolerant way, to gene therapy delivered using what has become the preferred gene delivery vector, adeno-associated viruses (AAV). A new review that takes a comprehensive look at both the AAV-mediated immune reactivation response to gene transfer and the role that regulatory and exhausted T cells play in the development of tolerance to AAV is published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers." The article is available free on the Human Gene Therapywebsite until May 27, 2017.

Gwladys Gernoux and Christian Mueller, University of Massachusetts Medical School, Worcester, MA and James M. Wilson, Perelman School of Medicine, University of Pennsylvania, Philadelphia, highlight the latest research in the new area of biomedical discovery focused on immunologic responses to AAV and the immune mechanisms that regulate chronic infections. In the article entitled, "Regulatory and Exhausted T Cell Responses to AAV Capsid," the researchers discuss the complexity of the immune response to AAV, the broad variability in responses between individual patients exposed to AAV vectors, strategies in development to suppress an immune reaction to viral vector components, and the implications of this evolving research area to future developments in AAV-based gene therapy.

"Several groups have observed very long-term expression of rAAV vectors in humans even in the face of immune responses to the capsid. The Treg and exhausted T cell responses that this article discusses may be the key to explaining this property, which appears to be critical for the clinical success of AAV as a vector," 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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Research reported in this publication was supported by the National Institutes of Health under Award Numbers P01-HL131471, R01-DL098252, R24-Od018259, and R01-NS088689. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

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 Therapywebsite .

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. 140 Huguenot St., New Rochelle, NY 10801-5215 http://www.liebertpub.com Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

Mary Ann Liebert, Inc./Genetic Engineering News

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