Gene Technology

Research Leader: Dr Don Anson

The Gene Technology group is focused on the development of lentiviral vector mediated gene therapy for inherited
metabolic diseases. The group has developed its own lentiviral vector technology and has considerable expertise in the large scale production and purification of lentiviral vectors.

Diseases of interest are the lysosomal storage disease mucopolysaccharidosis type IIIA, methylmalonic aciduria and cystic fibrosis. Research in these diseases is currently based on the use of animal models to evaluate the efficacy of different approaches to gene delivery. The work is done in collaboration with Dr Sharon Byers (Head, Matrix Biology Lab, SA Pathology) for mucopolysaccharidosis type IIIA, Dr David Parsons (Pulmonary Medicine, Women's and Children's Hospital) and Dr Janice Fletcher, Dr Heidi Peters (Women's and Children's Hospital and The Murdoch Institute, respectively).

Research Priorities:

• Evaluation of lentiviral mediated gene therapy for inherited diseases using mouse models of human disease,
  namely:
  • Mucopolysaccharidosis type IIIA
  • Methylmalonic aciduria
  • Cystic fibrosis