Gene Therapy Research Unit
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The Gene Therapy Research Unit, a joint initiative with the Children's Medical Research Institute, focuses on the development of novel gene-based strategies for the treatment of genetic conditions affecting children. This is a tremendously exciting but challenging area requiring both laboratory and clinical research expertise.
The key challenge is to safely repair or replace faulty genes with healthy ones in a sufficient number of cells to achieve therapeutic benefit.
Our approach involves the use of cutting-edge gene delivery technologies (vectors) derived from viruses and begins with genetic repair of isolated cells growing in cell culture followed by treatment of disease in small animals such as mice.
The liver and bone marrow are two particularly promising targets for gene therapy as many childhood genetic diseases involve these tissues.
Exceptional progress is being made in all areas, but of particular interest is the Unit's work on metabolic liver disease, which it is on the cusp of being translated from the laboratory into treatment for children with the disease.
The Unit has previously reported success in curing mice with Ornithine Transcarbamylase (OTC) deficiency using gene therapy. The challenge currently is to ensure that the gene delivery strategy that the Unit has devised will be safe and effective in humans. It has developed a research pathway to test the delivery of the human OTC gene to human liver cells and established the necessary collaborative relationships needed to reach this goal. This project is well on the way to generating the data necessary to justify and underpin a human clinical trial proposal.