Université Laval

Purpose

There are about 7,000 different hereditary diseases each due to the mutation of a gene. These genes are expressed in different cells. The treatment of these diseases requires the delivery of a normal gene or of gene modifying agents to the appropriate cells, i.e. those involved in the disease. Adeno-associated virus (AAV) is currently the most commonly used virus for the delivery of these components. However, delivery by AAVs is not sufficiently effective and specific for effective treatment of many hereditary diseases. Thus, the objective of this project is to modify the AAVs to produce some variants, which are more specific and more effective. The team’s approach is to modify the proteins, which are at the surface of the AAVs to change their binding to the proteins, sugar and lipids at the surface of target cells. This project will generate new AAV variants able to infect more effectively various types of cells to improve the efficacy of gene therapy