Embryonic Stem (ES) cells have played an indispensable role in helping researchers understand the function of human disease genes and in creating mouse models of human disease. ES research has the potential to transform knowledge of human disease into the development of new classes of therapeutic agents, from novel drugs to cell-based therapies. This research project identifies key barriers in the current state of ES cell technology and proposes novel technologies to overcome these barriers. Over time, these technologies are expected to significantly impact biomedical research programs and biotech companies in Canada and thereby increase the rate at which new medical discoveries are moved into health care delivery.
Funding Partners: Genome Canada, Government of Manitoba, Government of Alberta, and the Allen Institute of Brain Science.
ES cells have played an indispensable role in the discovery of the function of human disease genes and in creating mouse models of human disease. The “knockout” mouse, for example, allows researchers to identify gene function by examining the effect of gene alteration in ES cells and in mice derived from them. This powerful approach allows scientists to create animal models of disease, which can be used to study the causes of human disease and potential treatment or intervention strategies. With this proven and very powerful discovery tool, the International Knockout Mouse Consortium was established through an integrated $100M worldwide effort that is rapidly and efficiently generating designed mutations in every gene in the mouse genome. The consortium is also making this resource freely available to the biomedical research community. This resource is expected to have a wide reaching and significant impact across diverse scientific disciplines of a magnitude similar to those realized by the sequencing of the Human Genome.
ES cells have many other applications. Particularly with human ES cells becoming more prevalent in research, ES cells have the potential to transform our knowledge of human disease and development and give rise to new classes of therapeutic agents – from novel drugs to cell-based therapies. Such studies are done only in cells and are approved by a national stem cell regulatory board to make certain legal and ethical aspects of using human stem cells are taken into consideration by an independent body.
To ensure Canada is strategically positioned to take full advantage of mouse and human ES cell resources, the scientists in this proposal identify several key barriers in the current state of ES cell technology and propose to develop novel technologies to overcome these barriers. The proposed technologies in this project will be developed using mouse ES cells, but can be applied by others to any model cell system. Each cutting edge technology is recently established in principle by the respective principal investigators and the current project application will allow a three-prong implementation strategy: development of the technology, validation of the technology, and delivery of the technology in a form that is readily accessible and immediately usable by the scientific community without specialized training. These enabling technologies are expected to significantly impact biomedical disease-focus research programs and biotech companies in Canada and thereby increase the rate at which new medical discoveries are moved into health care delivery.