Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA
- PDF / 384,059 Bytes
- 8 Pages / 610 x 792 pts Page_size
- 58 Downloads / 149 Views
BioMed Central
Open Access
Research
Lentiviral-mediated gene correction of mucopolysaccharidosis type IIIA Donald S Anson*1,2,3,4, Chantelle McIntyre1,2, Belinda Thomas1,5, Rachel Koldej1,2, Enzo Ranieri1,2, Ainslie Roberts1,2, Peter R Clements1,2, Kylie Dunning1,6 and Sharon Byers1,2 Address: 1Department of Genetic Medicine, Women's and Children's Hospital, Children, Youth and Women's Health Service, 72 King William Road, North Adelaide, SA 5006, Australia, 2Department of Paediatrics, University of Adelaide, SA 5005, Australia, 3Department of Biotechnology, Flinders University, GPO Box 2100, Adelaide, SA 5001, Australia, 4School of Pharmacy & Medical Sciences, University of South Australia, GPO Box 2471, Adelaide, SA 5001, Australia, 5Department of Respiratory and Sleep Medicine, Monash Medical Centre, VIC 3168, Australia and 6Department of Obstetrics and Gynaecology, University of Adelaide, SA 5005, Australia Email: Donald S Anson* - [email protected]; Chantelle McIntyre - [email protected]; Belinda Thomas - [email protected]; Rachel Koldej - [email protected]; Enzo Ranieri - [email protected]; Ainslie Roberts - [email protected]; Peter R Clements - [email protected]; Kylie Dunning - [email protected]; Sharon Byers - [email protected] * Corresponding author
Published: 16 January 2007 Genetic Vaccines and Therapy 2007, 5:1
doi:10.1186/1479-0556-5-1
Received: 14 November 2006 Accepted: 16 January 2007
This article is available from: http://www.gvt-journal.com/content/5/1/1 © 2007 Anson et al; licensee BioMed Central Ltd. This is an Open Access article distributed under the terms of the Creative Commons Attribution License (http://creativecommons.org/licenses/by/2.0), which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited.
Abstract Background: Mucopolysaccharidosis type IIIA (MPS IIIA) is the most common of the mucopolysaccharidoses. The disease is caused by a deficiency of the lysosomal enzyme sulphamidase and results in the storage of the glycosaminoglycan (GAG), heparan sulphate. MPS IIIA is characterised by widespread storage and urinary excretion of heparan sulphate, and a progressive and eventually profound neurological course. Gene therapy is one of the few avenues of treatment that hold promise of a sustainable treatment for this disorder. Methods: The murine sulphamidase gene cDNA was cloned into a lentiviral vector and high-titre virus produced. Human MPS IIIA fibroblast cultures were transduced with the sulphamidase vector and analysed using molecular, enzymatic and metabolic assays. High-titre virus was intravenously injected into six 5-week old MPS IIIA mice. Three of these mice were pre-treated with hyperosmotic mannitol. The weight of animals was monitored and GAG content in urine samples was analysed by polyacrylamide gel electrophoresis. Results: Transduction of cultured MPS IIIA fibroblasts with the sulpha
Data Loading...
