Researchers improve vbectors for delivering hFVIII gene therapy to treat Hemophilia A

March 31, 2017

New Rochelle, NY, March 31, 2017--A new study examined 42 combinations of promoters and enhancers for human factor VIII (hFVIII) gene expression to identify the optimal adeno-associated virus (AAV)-based gene therapy delivery vector constructs to take forward into development. Evaluation of the different combinations in mice that lack factor VIII demonstrated the significant and differing effects the vector components had on liver-specific expression of the hFVIII transgene, as reported in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Human Gene Therapy website until April 30, 2017.

James M. Wilson, MD, PhD, Director of the Gene Therapy Program, Department of Medicine, University of Pennsylvania (Philadelphia, PA) and Editor, Human Gene Therapy Clinical Development, and coauthors from the University of Pennsylvania Department of Medicine and School of Nursing, and Dimension Therapeutics (Cambridge, MA), developed and compared the different AAV vectors to overcome the challenge of delivering the relatively large hFVIII gene and to achieve therapeutic levels of factor VIII gene expression.

In the article entitled "Characterization of AAV-Mediated Human Factor VIII Gene Therapy in Hemophilia A Mice," the researchers also compared the levels of antibody generated against the various AAV transgene delivery vectors.

"Dr. Wilson's group and their colleagues at Dimension Therapeutics continue to improve the design of AAV vectors designed to treat the more common form of hemophilia, hemophilia A," 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. "These design improvements are crucial as the practical application of gene therapy for hemophilia progresses."
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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 and focused 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.

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 http://www.liebertpub.comPhone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101

Mary Ann Liebert, Inc./Genetic Engineering News

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