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Dr. Donald S. Anson,
Head, Gene Technology Unit, Department of Genetic Medicine
Co-head, Adelaide Cystic Fibrosis Gene Therapy Group

Dr. Anson obtained his B.Sc (Hons) in Biochemistry from Imperial College, London, his PhD from Institute of Animal Genetics, University of Edinburgh and did postdoctoral studies at the Sir William Dunn School of Pathology, University of Oxford, and the Department of Cancer Biology, Salk Institute, before moving to the Department of Chemical Pathology at the Women's and Children's Hospital in 1989.

The Gene Technology Unit is interested in developing lentiviral vectors and using them for gene therapy for cystic fibrosis and the mucopolysaccharidoses.

Current projects include optimization of lentiviral vector production systems, development of stable packaging cell lines for lentiviral vectors, large-scale processing/purification of lentiviral vectors, gene therapy for mucopolysaccharidosis type IIIA (MPS IIIA) and gene therapy for cystic fibrosis.

Lentiviral vectors

Lentiviral vectors show great promise in gene delivery vectors in that they are able to efficiently deliver genes to dividing and non-dividing cell populations/tissues in situ. We have developed a lentiviral vector system from HIV-1 using a variety of approaches to address concerns regarding safety. This project is continuing. In addition, we are developing procedures, reagents and processes for large scale production and bioprocessing of lentiviral vectors, all being important issues in developing these vectors for clinical application.

Gene therapy for mucopolysaccharidosis type IIIA

This project is a collaboration with the Matrix Biology Laboratory (Head, Dr. Sharon Byers).

Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder that results in a severe neurological phenotype as well as widespread, although milder, somatic symptoms. Currently we are developing gene therapy approaches to treat the somatic symptoms of MPS IIIA. These will then be adapted to treat the neurological disease.

The Adelaide Cystic Fibrosis Gene Therapy Group

This group is headed by Dr. David Parsons (Department of Pulmonary Medicine, WCH) and myself. The aim of the group is to develop lentiviral mediated gene therapy for lung disease. A variety of models and projects are underway aimed at understanding the underlying science and developing practical methods which will be applicable in the clinic.

Selected publications

Bielicki, J, Hopwood, JJ and Anson, DS. (1996). Correction of Sanfilippo A skin fibroblasts by retroviral mediated gene transfer. Human Gene Therapy, 7: 1965-1970.

Fuller, M. and Anson, DS. (2001). Helper plasmids for production of HIV-1 derived vectors. Human Gene Therapy, 12: 2085-2097

Limberis, M, Anson, DS, Fuller, M. and Parsons, DW. (2002). Recovery of airway cystic fibrosis transmembrane conductance regulator function in mice with cystic fibrosis after single dose lentivirus-mediated gene transfer. Human Gene Therapy, 13, 1961-1970.

Anson, DS. and Fuller, M. (2003). Rational development of a HIV-1 gene therapy vector. J. Gene Medicine, 5, 829-838.

Anson, DS. and Limberis, M. (2004). An improved b-galactosidase marker gene. Journal of Biotechnology, 108, 17-30.

Fuller, M. and Anson, DS. (2004). Can the use of HIV-1 derived gene transfer vectors for clinical application be justified? Current Gene Therapy, 4, 65-77.

Anson, DS. (2004). The Use of Retroviral Vectors for Gene Therapy- What are the risks? A Review of Retroviral Pathogenesis and Its Relevance to Retroviral Vector Mediated Gene Delivery. Genetic Vaccines and Therapy, 2: 9.

Anson, D.S. and Dunning, K. (2005). Optimized expression constructs for HIV-1 Minor Proteins. J. Biotechnology, accepted for publication.

Anson, DS., Koldej, R. and Dunning, K. (2005). Optimisation of a Multipartite Human Immunodeficiency Virus Based Vector System. J. Gene Medicine, accepted for publication.

Anson, DS., Thomas, B. and Byers, S. (2005). Lentiviral mediated gene therapy for mucopolysaccharidosis type IIIA. In preparation.

Contact:

Associate Professor Donald S. Anson
Head, Gene Technology Unit
Department of Genetic Medicine
4th Floor Rogerson Building
Women's and Children's Hospital
Children, Youth and Women's Health Service
72 King William Road
North Adelaide  Sth Aust  5006
Australia

Telephone: +61 (0)8 8161 6373
Facsimile: +61 (0)8 8161 7100
Email: donald.anson@adelaide.edu.au