About the Cell & Gene Therapy Center
The Cell & Gene Therapy Center ("CGTC") was founded in 2001 at Cooper Hospital and the former UMDNJ-School of Medicine, as a combined clinical and basic research facility. The focus of our work is on neurological diseases that can be treated using the latest gene transfer technologies and cell transplantation methods. CGTC is a "translational" research facility, which means that we bring discoveries from the laboratory bench directly to the clinical setting.
The CGTC aims to identify potential therapeutic avenues arising from the characterization of models of human neurological diseases. The translation of basic research into therapy requires the investigation of disease mechanisms at multiple levels, ranging from the molecular to the behavioral. Investigators at the CGTC therefore adopt a multidisciplinary approach to the study of diseases of the brain and employ a wide range of laboratory techniques in this process.
The former UMDNJ and Cooper Hospital have hosted a NIH-sponsored Phase I/II study on Gene Therapy for Canavan Disease, in collaboration with Children’s Hospital of Philadelphia and The University of North Carolina, Chapel Hill, under a major grant from the National Institute for Neurological Diseases and Stroke (NINDS). This clinical trial represented the first use of adeno-associated viral vectors (AAV) in the human brain, as well as the first viral gene therapy for a neurodegenerative disorder.