In vivo gene transfer: focus on the kidney.

Renal gene transfer techniques are being developed as a novel experimental approach to understand the pathogenesis of renal disease and to potentially develop new therapeutic tools. We review the currently available technology to introduce foreign genetic material into renal tissue, i.e., retroviral, adenoviral, and liposomal transfer systems with their respective advantages and caveats. Today, the transfer efficiency of these methods appears to be sufficiently high to study the effects of transduced genes on renal function and morphology in rat kidney. This will allow (i) the elucidation of the function of genes on the course of renal disease in experimental animal models and (ii) the modulation of local expression of endogenous genes which presumptively contribute to renal pathology in these models. One strategy to accomplish this aim is the use of recombinant DNA technology to design antisense DNA constructs or oligonucleotides, which interfere with the renal expression of target genes. We will also discuss some of the shortcomings of the currently used techniques with respect to potential therapeutic use of gene transfer systems and gene modulation.