Gene Therapy for a genetic cardiovascular disease: AAV-mediated gene transfer of a powerful, naturally occurring, LPL-S447X variant for the treatment of LPL deficiency

Principal Investigator: 
University: 
University of British Columbia
Award Type: 

Dr. Colin Ross believes that studying genetics and diseases at the molecular level can open many doors for the treatment of diseases at their root causes. He's doing exactly that in cutting edge research to develop treatments for a genetic cardiovascular disease that has the highest worldwide frequency in Canada's French-Canadian population. People with lipoprotein lipase (LPL) deficiency are missing a key enzyme that helps break down triglycerides (fats) in the blood stream. Elevated levels of these fats can cause serious, life-threatening damage to the pancreas, heart and other organs. Ross is working on the development of gene therapy techniques to implant healthy genes into cells to restore production of the missing enzyme. He ultimately aims to develop a safe and long-term treatment for LPL deficiency.

Research Pillar: 
Host Institution: 
University of British Columbia
Research Location: 
Children's & Women's Health Centre of British Columbia
Co-Supervisor: 
Michael R Hayden
Year: 
2001