Gene delivery transplantation

DNA binding moiety has been synthesized and evaluated as a receptor-mediated gene delivery system. The system was able to transfect about 30% of corneal endothelial cells of rabbit, pig and human in the presence of chloroquine (Shewring et al., 1997). Associated with the fusogenic peptide of influenza, this peptide transfected 25-30% of primary cultures of vascular smooth muscle cells of man, rabbit and rat (Collins et al., 2000 Li et al., 2000). The molossin-based gene delivery system represents an interesting system in transplantation because the molossin peptide does not bind to vascular endothelium and pancreatic islets. 

Li, J.-M., Collins, L., Zhang, X.-H., Gustafsson, K. and Fabre, J.W. (2000) Efficient gene delivery to vascular smooth muscle cells using a nontoxic, synthetic peptide vector system targeted to membrane integrins a first step toward the gene therapy of chronic rejection. Transplantation, 70, 1616-1624. 

The use of HVJ liposome gene delivery for clinical trials has been evaluated in several animal models, and we believe that this gene delivery system is promising for the treatment of cancers, chronic diseases, and organ transplantation [19]. 

Kapturczak, M. H., Flotte, T. and Atkinson, M. A. (2001). Adeno-associated virus (AAV) as a vehicle for therapeutic gene delivery Improvements in vector design and viral production enhance potential to prolong graft survival in pancreatic islet cell transplantation for the reversal of type 1 diabetes. Curr. Mol. Med. 1, 245-258. 

Larkin DF, Oral HB, Ring CJ, Lemoine R, George AJ. Adenovirus-mediated gene delivery to the corneal endothelium. Transplantation 1996 61 363-370. 

Long-term expression, systemic delivery, and macrophage uptake of recombinant human glucocerebrosidase in mice transplanted with genetically modified primary myoblasts. Hum. Gene Ther. 9, 2375-2384. 

Opie SR, Dib N, Local endovascular delivery, gene therapy, and cell transplantation for peripheral arterial disease, J Endovasc Ther 2004 I I (suppl 2) 11151 —11162. 

The cloning of the factor VIII gene and development of retroviral-vector delivery systems have raised the possibility that the defect in haemophilia A may be corrected by gene therapy. In a sense this is already a reality patients with haemophilia A who underwent liver transplantation for progressive hepatic disease were found to be producing haemostatic concentrations of factor VIII. 

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