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A million dollar commitment from Jesse's Journey

Dr. Ron Worton, CEO of the Ottawa Hospital Research Institute who also discovered the Duchenne gene, says that "after 20 years in research on muscular dystrophy, it has become clear to me that the best hope for those with neuromuscular disorders is through regeneration of the damaged muscle and that stem cells provide the means of achieving this."

There is intense interest in biomedical research about "stem cells". These are immature forms of cells that are capable of transformation into many different kinds of mature cells -- that form blood, skin, organ, nerve and muscle.

Dr. Johnny Huard of the Pittsburgh School of Medicine has discovered a special population of stem cells derived from muscle tissue that show much promise in the treatment of genetic disorders -- notably Duchenne Muscular Dystrophy, which is the most serious of a wide range of disabling neuromuscular diseases. Turning his discovery into clinical treatment requires a tremendous amount of laboratory work and some unique systems of analysis and imaging.

The Duchenne Research Initiative brings together Dr. Huard's research team at the University of Pittsburgh, Pennsylvania, Dr. Michael Rudnicki's laboratory at the Ottawa Hospital Research Institute, and a team under the direction of Dr. David Hill at London's Lawson Health Research Institute. Each of these centers will receive funding from Jesse's Journey for special equipment and operations, in an unprecedented international collaboration seeking to turn stem cell discoveries into effective clinical treatment.

The University of Pittsburgh will acquire an extraordinary robotic cell analysis system that will culture large numbers of different cell populations and automatically record -- literally cell-by-cell -- the behaviour of each population. The system will monitor such vital factors as cell division, the ability to differentiate into other cell types, and the ability to express proteins, which are the tools of life itself at the cellular level.

The Ottawa Hospital Research Institute will be funded for equipment for the analysis of gene expression in candidate stem cell populations, including those referred from Pittsburgh. This work will provide enhanced understanding of the genes that regulate stem cell function and characterize promising cell lines, for use in Duchenne muscular dystrophy. Together these studies will provide insights into the biology of muscle regeneration. In the future, identification of gene targets has the potential to lead to the development of drugs that would ameliorate the progression of muscular dystrophy by stimulating the inherent regeneration capacity of skeletal muscle.

The Lawson Health Research Institute will be funded for the operation of its unique small animal imaging systems. It will monitor and assess the effectiveness of cell lines identified by the other two labs and implanted into small animal models. Lawson's facilities include Magnetic Resonance Imaging (MRI), Computer Tomography (CT) scanning and Positron Emission Tomography (PET) scanning -- all adapted to study the behaviour of implanted cells in living animal models. The London team will also translate its animal imaging into clinical applications for human patients, since the imaging technologies differ only in scale.

Jesse's Journey, The Foundation for Gene and Cell Therapy, is delighted to have contributed to the bringing together of the vision, skill and dedication of three great research teams and looks forward with enthusiasm to their achievements and the future expansion of this initiative. The Duchenne Research Initiative is dedicated to the many thousands, in Canada and worldwide, who support the Journey.

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