Vancouver (September 9^th, 2004) - Scientists have developed a promising new treatment for a blood disease using a virus to transfer a "super" gene. In the paper, to be published September 10th Human Gene Therapy, they have shown that this "super" gene therapy can deliver long-lasting treatment in mice with Lipoprotein Lipase Deficiency - a discovery that could lead to treatments for humans suffering from the disease.

Lipoprotein Lipase Deficiency is a severe genetic disease affecting children and has the world's highest incidence in Canada due to early settlers who happened to carry the gene at a higher frequency. Children with this disease do not produce lipoprotein lipase (LPL) properly leading to a dramatic accumulation of fats and cholesterol in the blood, symptoms of intense chronic abdominal pain and potentially lethal pancreatitis.

Investigators from UBC's Centre for Molecular Medicine and Therapeutics (CMMT) in collaboration with The Netherland's Academic Medical Center (AMC) and Amsterdam Molecular Therapeutics (AMT) used a naturally occurring beneficial variant of the LPL gene termed a "super" gene to treat mice with LPL deficiency. This "super" LPL gene is carried by 20% of the general population, who have lower than average fat and cholesterol levels in the blood and is much more effective at correcting the disease than the normal gene.

Mice that received a single dose of a non-pathogenic human virus carrying the "super" gene showed dramatically reduced fat accumulation in their blood to normal levels for over a year. "Using gene therapy to give individuals with Lipoprotein Lipase Deficiency the ability to naturally produce their own constant levels of LPL could potentially protect patients from the long-term debilitating effects of their disease", says Dr. Michael Hayden, Director of CMMT and Scientific Director of the Canadian Genetic Diseases Network.

This same "super" gene therapy may also have implications on the treatment of other genetic diseases, including heart disease. Ten percent of the general population are partially LPL deficient with mild accumulations of fats and reduced levels of good cholesterol in their blood. These patients have up to a 2-20 fold increased risk of heart disease, which could potentially be reduced by restoring normal levels of LPL using LPL gene therapy. By exchanging the therapeutic gene (in this case, the LPL gene) for a different gene missing in other patients, this technique also provides hope for the treatment of numerous unrelated genetic diseases.

This work would not have been possible without the support of the Canadian Genetic Diseases Network (CGDN), the Michael Smith Foundation, the Canadian Institute for Health Research (CIHR), the BC and Yukon Heart and Stroke Foundation and the BC Research Institute for Children and Women's Health.

About CGDN

The Canadian Genetic Diseases Network is a not-for-profit corporation, committed to advancing Canada's scientific and commercial competitiveness in genetic research, and the application of genetic discoveries to prevent, diagnose, and treat human disease. To achieve its objectives, CGDN participates in three essential activities: facilitates and funds collaborative research in human genetics across Canada; educates emerging scientists to excel in human genetic disease research; and facilitates partnerships between industry (biotechnology, pharmaceutical, and diagnostic firms) and academia to translate research discoveries into therapies or diagnostic tests.