Pioneer Award recipients Marina Cavazzana and Adrian Thrasher recognized for advancing gene therapy to the clinic for immunodeficiency disorders

March 24, 2014

New Rochelle, NY, March 24, 2014--Marina Cavazzana, MD, PhD, Paris Descartes University, France and Adrian J. Thrasher, MD, PhD, University College London Institute of Child Health, UK, have been honored with the Pioneer Award for basic and clinical gene therapy for immunodeficiency disorders. Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers, is commemorating its 25th anniversary by bestowing this honor on the leading 12 Pioneers in the field of cell and gene therapy selected by a blue ribbon panel* and publishing a Pioneer Perspective by the award recipients

Dr. Cavazzana has been at the forefront of advances in treating life-threatening inherited diseases of the immune system with gene therapy, using a patient's own modified stem cells. She describes the translation of this work to the clinic and its ongoing advances and novel applications in the article "Hematopoetic Stem Cell Gene Therapy: Progress on the Clinical Front." The article by Dr. Cavazzana is available free on the Human Gene Therapy website at http://online.liebertpub.com/doi/full/10.1089/hum.2014.2504.

A pioneer of gene therapy in the UK, Dr. Thrasher has been at the leading edge of basic science research on the function of therapeutic genes for inherited disorders and the development of viral vectors to deliver them to affected patients. He has collaborated on gene therapy clinical trials targeting immunodeficiency disorders with groups in Europe and the USA.

"Cell therapy and gene therapy are advancing together to improve patient care," says Dr. Cavazzana. "We can expect to be able to rebuild a new immune system not only in primary immunodeficiencies but also in severe acquired clinical conditions (such as those in HIV-1-infected patients)."

"I've seen some very exciting times in the field, from the first evidence that biochemical defects can be corrected in vitro, to some remarkable clinical successes in patients with devastating diseases. I look forward with huge enthusiasm to the exciting developments on the horizon, which are likely to impact on more patients with an even wider range of disorders," says Dr. Thrasher.

"These pioneers contributed to the first real clinical successes of gene therapy through their work in inherited immune deficiency disorders," 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.
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*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.

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 at http://www.liebertpub.com/hgt.

About the Publisher

Mary Ann Liebert, Inc., publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in 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 at http://www.liebertpub.com.

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

Mary Ann Liebert, Inc./Genetic Engineering News

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