PUBLIC RELEASE DATE:

3-Sep-2014

Contact: Kathryn Ryan kryan@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

New Rochelle, NY, September 3, 2014Robin Ali, PhD, University College London, Jean Bennett, MD, PhD, Perelman School of Medicine, University of Pennsylvania, and William Hauswirth, PhD, University of Florida College of Medicine, are co-recipients of the Pioneer Award, recognized for their leadership and contributions to the field of gene therapy to treat retinal degeneration leading to blindness. 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 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. Ali, Professor of Human Molecular Genetics, led proof-of-concept studies demonstrating the feasibility of using gene therapy to repair photoreceptor defects in the eye and of using cell transplantation for retinal repair. He also had a pioneering role in the first clinical trial for inherited retinal degeneration.

Dr. Bennett, Professor of Ophthalmology, Cell and Developmental Biology, recalls her first experiences with molecular biology and gene transfer technology, acquired in the lab of Dr. W. French Anderson, known as "the father of gene therapy." She describes her developing career, including the decision to go to medical school and to focus her research on developing adeno-associated virus (AAV) gene therapy techniques for restoring vision to patients affected by retinal degeneration in her Pioneer Perspective article entitled "My Career Path for Developing Gene Therapy for Blinding Diseases: The Importance of Mentors, Collaborators, and Opportunities," available on the Human Gene Therapy website.

Dr. Hauswirth, Rybaczki-Bullard Professor of Ophthalmology, traces his involvement in the field of retinal gene therapy to his early interest in studying the interaction between light and biological molecules. He provides a historical perspective on the discovery of the gene mutations responsible for several of the most common inherited eye diseases and the advances in AAV gene therapy technology being developed and applied to deliver replacement genes. His Pioneer Perspective, entitled "Retinal Gene Therapy Using Adeno-Associated Viral Vectors: Multiple Applications for a Small Virus," is available on the Human Gene Therapy website.

"These groups brought forward the first convincing clinical results of in vivo gene therapy, which paved the way for the current renaissance we are seeing in the field," 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.

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Pioneer Award recipients Robin Ali, Ph.D., Jean Bennett, M.D., Ph.D., and William Hauswirth, Ph.D.