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sector_ico_Health_trans Human Health

Pleiades Promoter Project

048PLE
  • Project Leaders: Elizabeth Simpson
  • Institutions: University of British Columbia (UBC)
  • Budget: $10118123
  • Program/Competition: Large Scale Competitions
  • Genome Centre(s): Genome Canada
  • Status: Closed

Gene therapy delivers healthy genes to replace dysfunctional ones and has been tried worldwide with some tantalizing successes as well as disappointing failures in some human trials. This project looked to improve safety of gene therapy by targeting gene expression to specific cell types in the brain through newly developed promoters. Using bioinformatics, the project characterized genes expressed in the mouse brain, identifying the particular cells and regions of expression and then determining the regulatory regions in the DNA. The project generated 159 characterized human DNA promoters to drive DNA expression in defined brain regions of therapeutic interest. These promoters immediately enable greater specificity in gene therapy vectors, and the resource will benefit both basic research and future human gene therapy. This project builds on a previously funded project, A Quantitative and Comprehensive Atlas of Gene Expression in Mouse Development, and was continued in a subsequent project, CanEuCre – Genomic Resources Advancing Therapies for Brain Disorders.