Dr. Jim Hu is presently a Senior Scientist in the Physiology & Experimental Medicine Program at the Research Institute, The Hospital for Sick Children (SickKids). He is also a Professor in the Departments of Laboratory Medicine and Pathobiology and Paediatrics and is a member of the Graduate Faculty of the Institute of Medical Science, University of Toronto.
Dr. Hu received his PhD in Biology from Harvard University. His PhD studies on RNA polymerase and gene transcription were carried out under the supervision of Dr. Lawrence Bogorad in the Department of Cellular and Developmental Biology. After graduating, Dr. Hu came to SickKids for his postdoctoral training in RNA splicing with Dr. James D. Friesen in the Department of Genetics.
Dr. Hu serves as a member of the American Society of Gene Therapy Genetic Diseases Committee (2007-2010) and as a member of the Medical/Scientific Advisory Committee of the Canadian Cystic Fibrosis Foundation and Chair of its Research Subcommittee (2006-2009). He has also served several terms as a member of the CIHR Respiratory Committee.
Abstract：
Despite the early hype in gene therapy, there are still major obstacles to translating genetic discoveries into clinical applications. These obstacles include innate and immune barriers to vector delivery, toxicity of vectors and the lack of sustained therapeutic gene expression. To overcome these obstacles, my research team has been developing novel gene expression cassettes and gene therapy vectors to improve therapeutic gene expression and to reduce host immune responses against gene therapy vectors. In addition, we have been developing novel delivery methods to achieve robust levels of transgene expression in small and large animal models. We have been using cystic fibrosis lung disease as an example to demonstrate the safety and efficiency of our technology. Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Although multiple organs and tissues are affected in CF patients, the major mortality and morbidity are due to lung failure. Gene therapy became an attractive therapeutic strategy because the lung airway is accessible to gene vectors. We have shown that our gene delivery system can be used to deliver human CFTR gene to CF knockout mice to protect them from acute lung infection. We have developed a strategy for vector re-administration through transient immunosuppression of the host immune system. We have recently shown that our vector system and delivery methods allow safe and effective delivery of genes to the pig lungs. These techniques can be adapted for gene therapy targeting other diseases, such as cancers.
