Public Release:  Faster, scalable method for producing AAV-based gene transfer vectors

Described in Human Gene Therapy

Mary Ann Liebert, Inc./Genetic Engineering News

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Credit: © 2010 Mary Ann Liebert Inc., publishers

New Rochelle, NY, January 5, 2011--A new, simplified method for producing large amounts of viral vector cassettes capable of shuttling genes into host cells will help advance the promising field of gene therapy as applications move into large animal studies and human clinical trials. The novel adeno-associated virus (AAV) production method is described in an article published Instant Online ahead of publication in Human Gene Therapy, a peer-reviewed journal published by Mary Ann Liebert, Inc. (www.liebertpub.com). The article is available free online at www.liebertpub.com/hum

This new method for creating stable AAV2 producer cell lines overcomes the major drawbacks of the previous method: it requires only a one-step cloning process (instead of multiple cloning steps), and a single (instead of two-stage) transfection and selection step to produce cell lines containing the circular, AAV vector-containing plasmid DNA needed to transport a gene of interest into host cells.

Zhenhua Yuan, Chunping Qiao, Peiqi Hu, Juan Li, and Xiao Xiao, researchers at the Eshelman School of Pharmacy, University of North Carolina at Chapel Hill, state that in addition to being faster and easier, their simplified method offers other important advantages, including the potential to produce high yields of multiple AAV serotypes upon co-infection with a helper adenovirus, as well as efficient packaging of single- or double-stranded AAV vectors and of large AAV cassettes. The authors describe their method in the article entitled, "A Versatile AAV Producer Cell Line Method for Scalable Vector Production of Different Serotypes."

"AAV gene therapy is showing promise in the clinic. Important challenges exist for it to become a commercial product such as scalable manufacturing processes. The study by Yuan et al. is an important step toward the development of a method of production that is capable of supporting a commercial product," says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania School of Medicine, Philadelphia.

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Human Gene Therapy, the Official Journal of the European Society of Gene and Cell Therapy, British Society for Gene 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 that 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. Tables of content and a free sample issue may be viewed online at www.liebertpub.com/hum

Mary Ann Liebert, Inc (www.liebertpub.com), 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 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 60 journals, books, and newsmagazines is available at www.liebertpub.com.

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

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