Dr. Paola Leone is an Associate Professor of Neurosurgery and Director of the Cell & Gene Therapy Center (CGTC). As Principal Investigator at UMDNJ-Robert Wood Johnson Medical School for Gene Therapy for Canavan Disease, she leads all research activities at UMDNJ and the affiliated centers. Her research interests have included in vivo neurochemistry of epilepsy and gene transfer approaches for the treatment of neurological disorders. She has published extensively in the field of CNS gene therapy and conducted the first direct gene therapy trial using a liposome-based vector for a pediatric neurodegenerative disorder. In addition to applications of viral vectors and stem cells, Dr. Leone is studying pharmacological approaches in humans and in animal models of Canavan Disease, Amyotrophic Lateral Sclerosis, Tay Sach’s and other neurological disorders.

Publications

Mitra Assadi, M.D.

Dr. Mitra Assadi initially worked at the NIH as a Research Fellow before starting her training in neurology in 1993 at Drexel University in Philadelphia. Upon completing her training in adult neurology in 1997, she did a fellowship in pediatric neurology at the St. Christopher Hospital for Children in Philadelphia. For the past three years, she has been working in Cooper hospital/UMDNJ as an Assistant Professor of Neurology. She is the clinical principal investigator of the lithium and buspirone studies.

Publications

Dr. Larissa Bilaniuk is a Professor of Radiology at the University of Pennsylvania School of Medicine and The Children's Hospital of Philadelphia (CHOP). She is the Principal Clinical Investigator of the clinical gene therapy study funded by a subcontracting grant from the National Institute of Neurological Disorders and Stroke. Dr. Bilaniuk has 15 years of experience as a clinical radiologist at CHOP. She is responsible for the coordination of the study at CHOP as well as the MRI reading during the screening of the patients and during the post-gene transfer follow-up tests.

Ms. Melissa Casas is an Administrative Assistant and Gene Therapy Coordinator for the Center. She assists with patient appointments and follow-up for all clinical studies, as well as other administrative duties.

Dr. Rick Cohen is a Research Scientist at the Center and the Coriell Institute for Medical Research, with a focus on developing techniques for affecting the differentiation of adult stem cells, and their use in therapeutic applications.

Publications

Michael Feely, M.D.

Dr. Michael Feely is a Professor of Surgery at UMDNJ-Robert Wood Johnson Medical School and a Pediatric Neurosurgeon at Cooper Hospital. Dr. Feely is the principal neurosurgeon of the clinical gene therapy study for Canavan Disease. He has extensive clinical experience in both pediatric and adult neurosurgery. He is trained in minimally invasive neurosurgical techniques, including the use of stereotactic targeting and real-time intraoperative imaging. Dr. Feely is overseeing the comprehensive operative and post-operative care of the Canavan patients.

Publications

Jeremy Francis, Ph.D.

Dr. Jeremy Francis is a post-doctoral fellow at the Center with an interest in signal transduction mechanisms relevant to a range of neurological diseases and processes. He is currently involved in the characterization of molecular aspects of brain development relevant to the Canavan Disease phenotype, which aims to identify the fundamental regulatory mechanism responsible for disease progression in affected individuals. He is also investigating aspects of the immune response in the brain with application to neurodegenerative autoimmune disorders.

Publications

Dr. Andrew Freese is a Senior Clinical Fellow of the Center. He serves as a research and clinical advisor on our gene therapy studies. Dr. Freese is the Vice Chairman of Neurosurgery at the University of Minneapolis.

Olga Goldfarb, M.D.

Dr. Olga Goldfarb is a pediatric neurologist with over thirty years of clinical experience, and she is an Assistant Professor of Pediatrics at the Children’s Regional Hospital at Cooper Hospital/University Medical Center. She conducts outpatient assessments on patients enrolled in ongoing clinical trials. She is also working to adapt quantitative neurology testing to the Canavan population.

Publications

Dr. Christopher Janson is an Assistant Professor in the Divison of Neurosurgery and Adjunct Assistant Professor in the Department of Molecular Genetics. As Principal Investigator of Gene Therapy for Canavan Disease at Cooper Hospital, he works on clinical aspects of that study and also conducts research on lysosomal storage disorders and Alzheimers disease. Dr Janson was a recipient of a 2003 NORD/Roscoe Brady Lysosomal Storage Diseases Fellowship Award.

Scott McPhee, Ph.D., (Adjunct)

Dr. Scott McPhee is a post-doctoral fellow at the Center with an interest in translational research for neurological disorders. He is currently working on the testing of novel gene transfer vector designs with in vivo models as part of the process of bringing new advancements in virology and molecular biology to the clinical setting. Dr. McPhee liases a close partnership with the UNC Gene Therapy Center for the optimization of clinical recombinant Adeno-associated viral vectors. With a background in neuroscience he is also working to identify the key mechanisms underlying the pathophysiology of Canavan disease, with likely significance to the study of numerous other neurodegenerative conditions. Dr McPhee was the recipient of the 2004 Canavan Clinical Research Training Fellowship, awarded by the American Academy of Neurology Foundation in association with the Canavan Foundation.

Michael L. Moeller, Ph.D.

Michael L. Moeller is an Associate Director of Surgical Research in the Department of Surgery at Cooper University Hospital. During his doctoral training, he was active in several joint academic/industry projects involving tissue engineering, wound healing, and stem cell biology. Recently, he served as a co-director of the Coriell Institute’s Stem Cell Training Course. His interests include the development of novel therapeutic tissue repair strategies using tissue-derived stem cells, the basic biologies of tissue-derived stem cells, and the molecular mechanisms of normal human development and homeostasis.

Publications

Ana Olariu, Ph.D., (Adjunct)

Dr. Ana Olariu is a Research Teaching Specialist at the Center and the focus of her work is stem cell therapies for neurodegenerative disorders. During a postdoctoral fellowship at the National Institutes of Health (NIH) she worked with Dr. Heather Cameron on adult neurogenesis and studied endogenous stem cells in the adult brain. She is currently working to develop a preclinical stem cell therapy for children affected by the Canavan disease. Having previously worked with Alzheimer’s disease animal models, Dr. Olariu is also interested in the role of neurogenesis in adult brain in the context of Alzheimer’s disease. Dr. Olariu was the recipient of the Fellow Award for Research Excellence (FARE) at the NIH in 2004.

Jude Samulski Ph.D., (Adjunct)

Dr. R. Jude Samulski oversees the clinical vector production for the Canavan Gene Therapy Study and serves as the Principal Investigator at UNC on a subcontracting grant from the National Institute of Neurological Disorders and Stroke. Dr. Samulski has over 20 years experience of engineering viruses for use as gene therapy vectors to deliver recombinant DNA. He is also a self-recognized authority on the live capture of copperhead snakes.

Evan Snyder, M.D., Ph.D. (Adjunct)

Dr. Evan Snyder is world renowned researcher in the stem cell field. His collaborative effort with our team focuses on understanding the mechanisms underlying Canavan disease and other childhood neurodegenerative diseases. Dr.Snyder's work investigates Canavan disease at a molecular and cellular basis.