James Ellis completed his B.S. at McGill University and his Ph.D. at the University of Toronto with Alan Bernstein, developing retrovirus vectors for gene targeting. His postdoctoral fellowship studying the beta-globin locus control region was mentored by Frank Grosveld in London, UK. Ellis established his own research team at the Hospital for Sick Children in Toronto in 1994 with a focus on gene therapy for sickle cell anemia. His first goal was to generate safe and effective retrovirus and lentivirus vectors for manipulating stem cells for molecular medicine. Stem cells silence viral vectors by compacting DNA into inaccessible chromatin structures.
Ellis’s group studied these silencing mechanisms and designed vectors with insulator elements that resist silencing. They developed MECP2 vectors for gene therapy of Rett syndrome and vectors with reporter genes that mark specific cell types. For example, their EOS (Early Transposon promoter and Oct-4 (Pou5f1) and Sox2 enhancers) lenti viral vectors express specifically in pluripotent stem cells and facilitate generation of induced Pluripotent Stem (iPS) cells from affected individuals. The Ellis team currently uses these iPSCs to model Rett syndrome, autism spectrum disorders and Williams Beuren syndrome. In addition, they perform drug screens on iPSC-derived cells to exploit their potential to discover personalized medicines. Ellis was appointed research integrity advisor at the Hospital for Sick Children in October 2013.