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Human MiniPromoters for Restricted Expression of Ocular Gene Therapy

Principal Investigator:
  • Elizabeth Simpson, University of British Columbia
Team Members:
  • Adriana Di Polo, CHUM Research Centre, University of Montréal
  • CQDM
  • Ontario Brain Institute

Project Overview

The purpose of this project is to develop promoters. Promoters are pieces of DNA, which act as switches to turn genes on and off. Such promoters are important tools for drug companies working on developing gene-based therapies; known as gene therapies. These are a type of biological therapy, somewhat like a vaccine, rather than a drug. Because such therapies are gene-based, they need promoters to turn them on and off; we argue, the better the switch, the safer and more effective the therapy. Blindness represents an enormous unmet disease burden in terms of human suffering and economic cost. Luckily, diseases of the eye are excellent targets for drug companies to develop gene therapies, and many companies are currently conducting clinical trials for such therapies in the United States. Thus, for this project, the promoters we are developing are all designed to control genes of the eye, and for therapies to treat blindness. For this project, the team is developing 30 new promoters for different cell-types in the eye, and thus useful for different diseases of the eye and different therapies for the eye. A major challenge is that normal genes have very large promoters, but for therapies the promoters need to be very small.