Overview

Microencapsulation is a cell-based method of gene therapy, using genetically-modified cells to provide a novel protein for the treatment of various inherited or somatic diseases. In contrast to conventional gene therapy using viruses to transfer therapeutic genes  into the patients’ own cells, this method uses  universal cell lines genetically engineered to secrete high levels of the therapeutic gene product. These cells are implanted within immuno-protective microcapsules into patients to act as a continuous source of the desired gene product, thus providing a potentially safer, reversible and more economic treatment than viral gene therapy.

Efficacy shown in mouse models of human diseases:

• Lysosomal Storage Diseases (MPS VII)
• Cancer (Anti-Angiogensis, Anti-Her2neu, Interleukin-2)
• Hemophilia (Factor IX Deficiency)
• Hormone deficiency (Dwarfism)

Our research is now focused on translating this technology to human application by 1) enhancing the physico-chemical properties of the biomaterials used to fabricate the microcapsules and 2) demonstrating efficacy in large animal models, especially in neurodegenerative lysosomal storage diseases.

Research groups  at McMaster University:

• Dr. Murray Potter   (Neurodegenerative lysosomal storage disease)
• Dr. Gonzalo Hortelano (Hemophilia)
• Dr. Harald Stover (Biomaterial)