How immune response differs for natural AAV infection compared to AAV vector for gene transfer?


New Rochelle, NY, August 10, 2016–A new, long-term study examined the antibody response to natural infection with adeno-associated virus (AAV) in chimpanzees for the purpose of characterizing the broad-based immune responses that could reduce the effectiveness of AAV vector-based gene delivery strategies. The study, which demonstrated the production of antibodies able to cross-neutralize multiple AAV serotypes, is published in Human Gene Therapy Clinical Development, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website until September 9, 2016.

Coauthors Roberto Calcedo and James M. Wilson, University of Pennsylvania Perelman School of Medicine, Philadelphia, monitored a group of chimpanzees — chosen because of their genetic similarity to humans — for 10 years and measured the levels of circulating antibodies in response to infection with naturally occurring AAV. The authors discuss the difference observed in the immune response to natural AAV infection compared to administration of AAV vectors used to deliver gene therapy in the article entitled "AAV Natural Infection Induces Broad Cross-Neutralizing Antibody Responses to Multiple AAV Serotypes in Chimpanzees".

"The impact of anti-vector antibodies remains a technical hurdle in systemic applications of AAV gene therapy," says Human Gene Therapy Clinical Development Editor James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia, PA. "The study of chimps provided us a window into the type of antibody response that occurs following a natural AAV infection."


Research reported in this publication was supported by the National Institute of Child Health and Human Development under Award Number P01-HD057247, the National Heart, Lung, and Blood Institute under Award Number P01-HL059407, and the National Institute of Diabetes and Digestive and Kidney Diseases under Award Number P30-DK047757. 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 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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