Some two million Canadians live with a genetic disease. Many of these diseases are not well understood and treatment and care is a challenge; existing treatments are often prohibitively expensive, imposing costs beyond the capacity of the health care system. Gene therapy has the potential for treating numerous genetic diseases. The concept is simple: gene therapy delivers and activates a gene to replace an abnormally functioning gene, thereby restoring the function of that gene. Its application is more complicated, in part because most current therapies do not restrict activation of the delivered gene to affected cells only, potentially resulting in undesired side effects.
Dr. Wyeth Wasserman of the University of British Columbia is leading a team to develop OnTarget, a software application that will allow scientists to design short DNA sequences to specifically activate delivered genes in targeted cells only. Through a user-friendly web-interface, scientists will be able to select and optimize human DNA sequences to target therapeutic gene expression to the right cells for their disease of interest. OnTarget will reduce the costs of designing gene therapy treatments with the potential to increase safety and efficacy. The ultimate beneficiaries will be patients with genetic disease in need of treatment.