pEPI for Gene Therapy Non viral episomes and their Application in Somatic Gene Therapy

Abstract

Nowadays, in most gene therapy trials virus based vectors are used because of their high efficiency. Nevertheless, safety risks like transformation of the cell by viral proteins, insertional mutagenesis, or innate immune reactions cannot be excluded. Basing on the idea of an ideal vector for gene therapy that is highly efficient but lacks these safety risks, non viral vectors systems represent an attractive alternative. With the construction of the non viral, S/ MAR based vector pEPI at the end of the last century, a first step towards non viral gene therapy has been made. S/MAR based vectors do not contain any viral elements, do not integrate and show stable transgene expression in the targeted cell or organism. Within the last decade, S/MAR based vectors were further improved and modified, and find now broad application in basic research and also become more and more recognized in gene therapeutic and clinical trials.