The research funding for the Stem Cell Network will support a number of national projects, including an effort led by Dr. Jacques Galipeau of the Montreal-based Jewish General Hospital to study the use of adult stem cells as repair material to help patients with cardiovascular and respiratory diseases.

Researchers are optimistic that stem cells, which can be found in fat, blood, bone and cells themselves, will eventually be used to repair damaged hearts, lungs and blood vessels.

Stem cells are like "seeds" and can be used "to grow a crop of new tissue," says Galipeau, which could be used to repair and restore damaged tissue. He wants to bring these technologies to real people. Scientists in Canada can use adult and embryonic stem cells for research but can't legally push the limits any further. Referring to the production and then destruction of week-old embryos to harvest stem cells, Galipeau says there is no therapeutic cloning allowed in Canada.

Later this spring, Galipeau and Toronto-based Dr. Duncan Stewart of St. Michael's Hospital will have patients in the late stages of the pulmonary arterial hypertension disease, who are awaiting life-saving transplants, take part in a trial that will attempt to combine stem cells and gene therapy to treat it.

Scientists from Montreal, Toronto, Vancouver and Ottawa would like to have clinical trials within the next two to five years with patients using their own stem cells to repair damaged hearts and lungs.

Cotler called it a "landmark day" for medical research and medical care, noting that Canada is among the leaders in stem cell research.

In this study, highly specific engineered ZFNs designed to bind to sequences close to an X-linked SCID-causing mutation in the IL2Rgamma gene resulted in a high percentage of the cells undergoing gene correction. In addition, it was observed that approximately one third of the corrected cells acquired the desired modification on both chromosomes. The expected downstream changes in both RNA and protein levels were also observed. Comparably high levels of correction were observed in primary human T-cells. While further work will be required to optimize the system for therapeutic use, the gene correction efficiencies established here may be sufficient to achieve a therapeutic effect.

Edward Lanphier, Sangamo's president and CEO, congratulated all of the Sangamo scientists involved in the generation of these data, and said it was gratifying that their work has been recognized by the prestigious journal Nature. These results highlight the potential for gene correction therapy for human monogenic disorders i.e. those diseases caused by mutation of a single gene. They are now working with their clinical collaborators to move the technology into the clinic. Initial research will focus on monogenic diseases of blood cells such as Sickle Cell Anaemia and beta-Thalassemia. The technology also forms the basis of their program to develop a potential therapeutic for HIV infection by disrupting expression of the CCR5 gene to generate a population of HIV-resistant cells.

This research is published in Nature as an advance online publication.

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