Gene Therapy for Glomerular Disease

In its broadest terms, gene therapy can be defined as the treatment of disease by transfer of genetic material into cells. The challenge in any organ system is to choose the correct gene to treat an individual's disease and to target that material specifically to the desired cell type. The Trojan horse vector to deliver that genetic material in solid organs is most commonly adeno-associated virus, modified to lose its replicative capacity. For the therapy to achieve the longevity of effect, the target cell should not divide because the transduced gene is episomal, rather than integrating into the host genome.