Does AAV-based gene delivery cause liver cancer? The debate heats up


New Rochelle, NY, May 26, 2016–Liver cancer can be triggered by mutations in cancer driver genes resulting from the insertion of adeno-associated virus (AAV) vectors used to deliver therapeutic genes, although this tumor-inducing role of AAV remains highly controversial. Recently published evidence of AAV-associated hepatocellular carcinoma was previously re-examined in Human Gene Therapy, and a new article in the Journal, published by Mary Ann Liebert, Inc., publishers, strongly challenges the re-interpreted data. The article is available free for download on the Human Gene Therapy website until June 26, 2016.

Jean-Charles Nault, Jessica Zucman-Rossi, INSERM, and coauthors, strongly disagree with the re-interpretation of their research, originally published in Nature Genetics, which appeared in a recent article by Kenneth Berns and colleagues in Human Gene Therapy. In the current article, "AAV2 and Hepatocellular Carcinoma", Nault et al. reaffirm their findings that insertional mutagenesis caused by AAV2 gene delivery vectors contribute to a subset of liver cancer cases in rare patients. The authors also state, "we fully disagree with Berns and colleagues, who claimed a protective role for AAV infection after re-interpreting our results," emphasizing that there is no good evidence to support a tumor suppressive effect of AAV2 in human liver cells or human cancers in general.

"Our ultimate goal as translational scientists is to develop new therapies that are both safe and effective," 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. "It is crucial that scientists can engage in such vigorous debates, which in turn generate interest in future studies to clarify whether or not rAAV-based gene therapy holds significant cancer risk to patients. We are pleased that Human Gene Therapy can provide the forum for such debates."


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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