Proposed link between liver cancer and adeno-associated virus challenged in human gene therapy



IMAGE: 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…

Credit: ©Mary Ann Liebert, Inc., publishers

New Rochelle, NY, January 6, 2016–The conclusion drawn from a recent study that insertion of adeno-associated virus 2 (AAV2) into human DNA causes mutations leading to the development of hepatocellular carcinoma (HCC) was resoundingly rejected by leading researchers in the fields of gene therapy and molecular genetics. Calling the conclusions of the study authors "an enormous leap from their data," the team of researchers challenge details of the experimental methods, interpretation of the findings, and limitations of the study design in an Editorial published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers . The Editorial is available free to download on the Human Gene Therapy website until February 6, 2016.

"Adeno-Associated Virus Type 2 and Hepatocellular Carcinoma?" is coauthored by Kenneth Berns, Barry Byrne, Terence Flotte, Guangping Gao, William Hauswirth, Roland Herzog, Nicholas Muzyczka, Thierry VandenDriessche, Xiao Xiao, Sergei Zolotukhin, and Arun Srivastava.

Challenging a recent Letter in Nature Genetics by Nault et al., the authors of the HGT Editorial instead suggest that "AAV infection might indeed be a key factor in preventing HCC in humans." Up to 90% of humans have AAV2 in their blood, yet HCC affects only about 10/100,000 people in the U.S.

In the Editorial, the authors assess the study design and data. They conclude that in most cases, AAV2 present in the DNA of liver cells from patients with HCC either slowed or had no effect on tumor growth, as it was detected in only 7% of HCC tumors and in 21% of adjacent normal liver tissue samples.


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 content 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, Genetic Engineering & Biotechnology News (GEN), 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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