GENE-THERAPY AND THE KIDNEY - BASICS AND IMPLICATIONS

The concept of gene therapy is simple: correcting genetic material of the target cells in order to cure diseases. Gene-mimetic substances ne ed to be delivered into the cells via gene carriers called vectors. Li mitations of vector systems still represent a major challenge to the s uccess of human gene therapy. The choice of gene therapy approach depe nds on the nature of disease and the accessibility of the target. In t he case of diseases, where the overexpression of defined genes causes functional damage, antisense oligonucleotides may represent an alterna tive to the conventional pharmaceuticals. Situation around the donor k idney prior to transplantation offers an ideal setting for gene therap y, which our group exploits in order to influence initial events which are associated with chronic graft dysfunction. This review will discu ss: principles of gene therapy and gene transfer; basics of viral and non-viral vectors; antisense oligonucleotide technology; and gene ther apy for reperfusion injury in renal transplantation.