NGF Medical Board

Dr. John Barranger
University of Pittsburgh
Graduate School of Public Health
Department of Human Genetics
21 Wilson Dr
Pittsburgh, PA 15202
Tl: (412) 734-4672
Fx: (412) 734-5476
E-mail: john.barranger@mail.hgen.pitt.edu

Dr. John Barranger is a professor of Human Genetics at the University of Pittsburgh. In 2004, he increased consulting activities to expand his role in Gaucher disease and other lysosomal diseases. In this consulting work, he collaborates with other scientists on cell and original models useful for improving existing, as well as, developing new therapies for lysosomal diseases. He is also involved in the planning and evaluation of a variety of clinical trials. In this consulting role, he travels extensively to train and educate. He evaluates testing laboratories and instructs laboratory directors and technicians on the execution of diagnostic studies for lysosomal disorders. In clinical outreach work, he provides clinical diagnostic and treatment education that impacts many hundreds of patients. In Pittsburgh, Dr. Barranger continues to advise at the University on patients with lysosomal disorders. Prior to 2004, Dr Barranger was also a professor in the Departments of Human Genetics, Molecular Genetics and Biochemistry, and Pediatrics at the University of Pittsburgh where he developed and directed the Human Gene Therapy Applications Laboratory, the Center for the Study and Treatment of Jewish Genetic Diseases, and the Comprehensive Gaucher Disease Treatment Center. He was co-director of the Human Gene Therapy Center, and Medical Director of the Molecular Medicine Institute. Previously, Dr Barranger was a scientist at the National Institutes of Health (NIH), where, from 1976 to 1978, he served in the Clinical Investigations and Therapeutics Section, Developmental and Metabolic Neurology Branch (DMNB), National Institute of Neurological and Communicative Disorders and Stroke. He ultimately became Chief of the Molecular and Medical Genetics Section of the DMNB and Associate Branch Chief. Dr Barranger received a PhD and MD from the University of Southern California Los Angeles, and completed an internship and residency in pediatrics at the University of Minnesota and a fellowship in Genetics at the NIH.

Dr Barranger has devoted his professional career to the study of inherited metabolic diseases, particularly lysosomal storage disorders. His research interests include biochemical and molecular genetics, enzyme therapy, gene transfer, and models of genetic disease. As a result of his investigation of the structure of glucocerebrosidase and receptors on macrophages, Dr Barranger developed successful enzyme replacement therapy for Gaucher disease, an inherited, disabling lysosomal disorder that affects more than 10,000 people in the United States. This work spawned the emergence of a new biotechnology industry which began with Genzyme Corporation. The first enzyme replacement therapy received Food and Drug Administration approval in 1991 and serves as a treatment model for more than 50 related lysosomal storage disorders. It is the paradigm of the practical accomplishments of translation research. He has published more than 450 papers and abstracts.

Included among Dr Barranger's honors are the March of Dimes Health Career Award, the United States Public Health Service Commendation Medal, the Arthur S. Flemming Award, and the Scientific Achievement Award of the National Gaucher Foundation. In addition to consulting for government and industry, Dr Barranger is a member of numerous professional organizations, including the Alpha Omega Alpha, American Society of Biochemistry and Molecular Biology, American Society of Human Genetics, Society for Inherited Metabolic Disorders, the Society for Pediatric Research, and the American College of Medical Genetics.

CV Highlights
Defined the structure of glucocerebrosidase
Invented the drug delivery system for glucocerebrosidase(GC)
Provided the proof of principle of ERT in the first patient
Cloned the gene for glucocerebrosidase
Described the most common mutations in the GC gene
Developed a commercial lab for LSD enzymatic and molecular diagnosis
Developed first LSD Treatment Center at a university