Gene therapy in transplantation: Toward clinical trials
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T. Ritter, M. Nosov & M.D. Griffin (2009), "Gene therapy in transplantation: Toward clinical trials", Current Opinion in Molecular Therapeutics 2009, 11:504-512
The genetic modification of organs or cells is an attractive approach to protect allogeneic transplants from acute rejection and other complications. The transplant setting offers a unique opportunity to utilise ex vivo gene therapy for modification of allogeneic organs and tissues prior to implantation. Significant challenges to applying this concept to human organ transplantation, however, include the large number of potential molecular targets, the diversity and safety profile of available vector delivery systems and the merging of gene-based therapies with existing immunosuppressive regimens. Accordingly many different therapeutic concepts and vector systems have been investigated in the pre-clinical area with a view to prolonging allograft survival but translation of promising gene therapy strategies to human clinical transplant studies has lagged behind the progress seen in other medical fields. In this article recent pre-clinical experimental applications of gene transfer to transplantation are outlined and the degree to which gene therapy has been clinically tested in organ transplant recipients is critically reviewed.