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Gene therapy pioneers Richard C. Mulligan and A. Dusty Miller reflect on their groundbreaking discovery

Mary Ann Liebert, Inc./Genetic Engineering News

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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... view more

Credit: ©Mary Ann Liebert, Inc., publishers

New Rochelle, NY, February 5, 2015--Recognized for their pioneering work in the development of gene transfer technology using retroviral vectors to deliver therapeutic genes into cells, Richard C. Mulligan, PhD, Director of the Harvard Gene Therapy Initiative, Harvard Institutes of Medicine, Boston, MA, and A. Dusty Miller, PhD, Fred Hutchinson Cancer Research Center, Seattle, WA, received the Pioneer Award from Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. Human Gene Therapy is commemorating its 25th anniversary by publishing a monthly Pioneer Perspective written by a leading pioneer in the field of cell and gene therapy selected for the Pioneer Award by a blue ribbon panel*. The Perspectives by Dr. Mulligan and Dr. Miller are available free on the Human Gene Therapy website.

Dr. Mulligan developed his interest in gene transfer technology and gene therapy while completing his undergraduate degree at MIT. During his graduate studies at Stanford University he and his colleagues developed some of the first DNA-based vectors to stably transfect cells with selectable markers. Convinced that viral vectors, and specifically delivery vehicles derived from retroviruses could stably introduce genes into a variety of cell types, Dr. Mulligan pursued this line of research during a postdoctoral fellowship at MIT. He enthusiastically describes the successful development of the first system for the production of "helper-free defective retroviral vectors" in his Pioneer Perspective entitled "Development of Gene Transfer Technology." Dr. Mulligan traces his long career in academic and translational research, refining gene transfer technology and moving retroviral vector-based gene therapy strategies to clinical testing. After serving as the chief scientific officer for the gene therapy company Somatix Therapy Corp. which he founded while at MIT, Mulligan has since moved his laboratory to Harvard, where he has dedicated his efforts to translational gene therapy activities and the development of vectors and reagents appropriate for clinical use.

In his Pioneer Perspective, "Retroviral Vectors: From Cancer Viruses to Therapeutic Tools," Dusty Miller describes how an undergraduate educational background in engineering and mathematics inspired him to focus on biology and complete a PhD in Pharmacology at Stanford University. Dr. Miller's work on retroviruses began during his postdoc at the Salk Institute, where he and colleagues demonstrated the feasibility of proper gene regulation following retroviral gene transfer. His search for a faculty position led him to the Fred Hutchinson Cancer Research Center in 1984, where early work in gene therapy was underway. Dr. Miller describes the evolution of his research, beginning with the development of a robust system for producing high concentrations of helper virus-free retroviral vectors to transfer and express a variety of genes in cells from multiple species including humans. The clinical utility of retroviral vectors to treat genetic diseases in humans has subsequently been demonstrated, and Dr. Miller is hopeful that continued improvements in the technology and protocols, overcoming regulatory challenges, and reductions in treatment costs will make it possible for gene therapy to be widely applied in the future.

"These pioneers essentially created the field of gene therapy through the development of retroviral vectors for genetically engineering cells," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia. "Their legacy goes well beyond the seminal scientific contributions they made to the field. Their true legacies are the second-generation scientists whom they educated, many of which are leaders in the field today."

*The blue ribbon panel of leaders in cell and gene therapy, led by Chair Mary Collins, PhD, MRC Centre for Medical Molecular Virology, University College London selected the Pioneer Award recipients. The Award Selection Committee selected scientists that had devoted much of their careers to cell and gene therapy research and had made a seminal contribution to the field--defined as a basic science or clinical advance that greatly influenced progress in translational research.

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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. 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 sister 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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