Project Overview

Gene therapy is the most promising new frontier in medical therapeutic intervention. Its premise is to deliver healthy genes to an organ to replace dysfunctional genes. Worldwide, numerous gene therapy trials have been conducted, some with devastating consequences, but some with tantalizing successes. From these experiences, several themes to increase safety have emerged: 1) more clinically relevant basic research is needed; 2) genes should be delivered to specific cell types to reduce the possibility of side effects from treating healthy cells and 3) genes should be delivered in a controlled manner to a specific location in the human genome, rather than allowing new genes to insert randomly – a situation that can be mutagenic. The Pleiades Promoter Project will use the latest scientific techniques to address all three.

One of the outcomes of the Project will be to use bioinformatics to characterize genes expressed in the mouse brain and identify which cells and regions they are expressed in and which segments of DNA (the promoters) turn the genes on and off. This data will provide an invaluable resource to scientist and clinical researchers. The end goal of this project is to generate 160 fully characterized, human DNA promoters of less than 4 kb (MiniPromoters) to drive gene expression in defined brain regions of therapeutic interest for diseases such as Alzheimer, Parkinson, Huntington, Amyotrophic Lateral Sclerosis, Multiple Sclerosis, Spinocerebellar Ataxia, Depression, Autism, and Cancer.

This Project is the daughter of an ongoing Genome Canada Project, the Atlas of Gene Expression in Mouse Development, within which the mouse brain gene expression data have already been gathered. The project also builds on three additional Genome Canada investments: the Bioinformatics of Mammalian Gene Expression developed software applicable to this work; the Genome BC Sequencing and Mapping Platform will provide sequencing; and the Genome BC Bioinformatics Platform will support the entire undertaking.

One of the strengths of The Pleiades Promoter Project is the way it brings together highly specialized Canadian resources. Bioinformatics, high throughput genomics, neuroimaging and transgenic mouse technology are all important activities on their own but together, they have the power to create success for Canada. The process of the Project will generate highly valuable resources that will gain high recognition for Canadian Scientists. To ensure the success of these resources (bioinformatics software, promoters, mouse embryonic stem cell lines, and transgenic mice), the team has collaborated with International BioPharma Solutions Ltd., a management and communications consulting company specializing in product development and commercialization advice.

Finally, the project will explore the challenging interface between science and journalism with a focus on genomics and gene therapy. A team lead by Dr. Stephen Ward of the UBC School of Journalism will develop new ways to better communicate controversial science to the public.