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Gene therapy for liver disease advancing with the help of adeno-associated viral vectors

December 30, 2016

New Rochelle, NY, December 30, 2016--Liver-directed gene therapy delivered using adeno-associated viral (AAV) vectors to treat diseases such as hemophilia have advanced into human testing. The potential for continued technological improvements to expand the therapeutic applications of gene therapy to treat liver disorders and the remaining clinical challenges are examined in a comprehensive review article published in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available open access on the Human Gene Therapy website.

In the article "Adeno-Associated Virus Gene Therapy for Liver Disease," Lisa Kattenhorn and coauthors from Dimension Therapeutics, Cambridge, MA, provide historical context for the remarkable progress achieved using this viral particular delivery vector to target therapeutic genes to the liver. The authors describe the preclinical and clinical studies that have led to a better understanding of immune responses to AAV gene therapy. In addition, they explore areas for future development and current challenges, including readministration of AAV gene therapy and minimizing the risk of hepatocellular carcinoma.

"AAV-based genet therapy to the liver has been a platform for transformational new therapies for genetic diseases such as hemophilia and inborn errors of metabolism," 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. "The review by Dr. Kattenhorn and colleagues provides an excellent overview of the current best knowledge in this area."
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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 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./Genetic Engineering News

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