|Dr. Yu-quan Wei
Yu-quan Wei, MD, PhD, Chairman and Professor, National Key Laboratory of Biotherapy and Cancer Center, West China Hospital, West China Medical School, Sichuan University, Sichuan, The People’s Republic of China. Interested in the development of gene and cell therapy, cancer vaccine, inhibition of angiogenesis, induction of apoptosis in tumor cells and clinical bio-chemotherapy.
Dr. Wolfe's research program investigates vector-mediated gene transfer and stem cell transplantation, including human iPS cell derived neural stem cells, to the brain in animal models of neuro-developmental genetic diseases of children. Studies are done in both rodents and domestic animal models with the same disease as humans. Domestic animals are a translational intermediates between mice and human patients to optimize procedures, methods and biological effectiveness of the treatment strategies. The research is directed towards increasing the distribution of a therapeutic protein, which is critical in genetic diseases because lesions in genetic diseases are typically distributed widely in the brain. Another important goal is to develop methods to monitor changes in pathology by non-invasive imaging modalities, as well as to track transferred genes or stem cells, which will be critical for clinical usage. Finally, although the genetics and biochemistry of the disease-causing genes are well understood for most neuro-developmental diseases, the actual mechanisms of neuropathology that result in intellectual disability remain obscure. A comprehensive approach is being used to understand the interrelationships between changes at the transcriptome, proteome, electrophysiological, and histopathological levels.
|Dr. Barry Byrne
Dr. Barry Byrne is a clinician scientist who is studying a variety of rare diseases with the specific goal of developing therapies for inherited muscle disease. As a pediatric cardiologist, his focus is on conditions that lead to skeletal muscle weakness and abnormalities in heart and respiratory function. His group has made significant contributions to the understanding and treatment of Pompe disease, a type of neuromuscular disease due to glycogen storage in motor units. The research team has been developing new therapies using AAV-mediated gene therapy to restore muscle function in Pompe and other inherited myopathies. His group at the Powell Center has also established a series of new methods for large-scale AAV clinical manufacturing.
He is the Associate Chair of Pediatrics and Director of the University of Florida Powell Center. He obtained his B.S. degree from Denison University, his M.D. and Ph.D. from the University of Illinois and completed his Pediatrics residency and cardiology fellowship as well as post-doctoral training in Biological Chemistry at the Johns Hopkins Hospital. He joined the University of Florida in 1997 and is now the Early and Christy Powell University Chair in Genetics.
|Dr. Jacques Tremblay
Dr. Jacques Tremblay is professor in the department of Molecular medicine of the Faculty of Medicine at Laval University in Quebec, Canada. He received the Best Researcher Award from Muscular Dystrophy Canada in 2005 and in 2006 the Henry Friesen Award presented by the Royal College of Physicians and Surgeons of Canada. He was a deputy editor for the Molecular Therapy journal form 2010 to 2013. He is the current president of the Association de Thérapie Génique du Québec (ATGQ). He is doing research to develop a cell therapy and gene therapy for Duchenne Muscular Dystrophy (DMD) and Friedreich ataxia. For this research, he is using various techniques including tissue culture, viral vector, ZFNs, TALENs and the CRISPR system. He has conducted a phase I clinical trial of myoblast transplantation for DMD. He has published 243 peer reviewed articles.
|Dr. Guangping Gao
Guangping Gao, Ph.D., is the Director of the Gene Therapy Center and Penelope Booth Rockwell Professor in Biomedical Research, UMass Medical School. He received his Bachelor Degree in Medicine from Sichuan University, China, and his Ph.D. in molecular genetics at Florida International University with his work on discovering the human aspartoacylase gene and the genetic mutations responsible for Canavan disease. Dr. Gao joined the Institute for Human Gene Therapy (IHGT) at University of Pennsylvania in 1994 and served as the Director of Vector Program of IHGT to oversee the vector discovery, process development, vector manufacturing and QC testing. One of his most important contributions to the field of gene therapy is the discovery of a novel family highly efficient and safe AAV vectors. His primary research interests include molecular mechanisms of AAV evolution, microRNA functional genomics in adult mammals, biology and clinical application of miRNA therapeutics, and gene therapy of CNS disorders using rAAVs to cross BBB for global CNS gene transfer. He has published more than 140 papers in peer-reviewed journals and has 26 patented inventions. Dr. Gao served on several international committees for gene therapy and editorial boards for journals. He is the senior editor of the Book Series on Gene and Cell Therapy publishing by ASGCT and Springer Publisher and serves on Advisory Board of Advances in Experimental Medicine and Biology.
| Prof George DICKSON
George Dickson is Professor of Molecular Cell Biology at Royal Holloway - University of London (RHUL). He has spent most of his career studying neuromuscular disease and muscle cell biology, including the first cloning of an intact dystrophin gene, the discovery of the role of cell adhesion molecules in muscle stem cell fusion, the first identification of utrophin, and the first description of exon skipping in Duchenne muscular dystrophy (DMD). Professor Dickson has also conducted notable research into gene therapy for atherosclerosis, and genetic vaccination against HIV/AIDS. He is a member of the UK MDEX and the UKHIV-Vaccine research consortia, and has been a platform leader in the EU Clinigene Network of Excellence. He is a past President of the European Society of Gene & Cell Therapy, and a past Secretary and founder member of the British Society for Gene Therapy. He has been a member of the European Medicine Agency Committee for Advanced Therapeutics (Gene and Cell Therapies). Present research is funded by EU, Muscular Dystrophy Charities, Welcome Trust, UK Department of Health, and the Bill & Melinda Gates Foundation, and includes: (i) Optimisation of exon-skipping and antisense oligonucleotide formulations for DMD, (ii) Inhibition of myostatin by RNA skipping and gene therapy for DMD, (iii) Development of dystrophin minigenes and AAV viral vectors for treatment of DMD, (iv) Direct genome correction of DMD using endonuclease-mediated genome surgery, (v) Development of adenoviral vaccine vectors for HIV/AIDS, (vi) General muscle fibre and stem cell biology.