Parvovirus-based vectors for human gene therapy.

Abstract

It is becoming increasingly clear that the parvovirus-based vectors may prove to be a useful alternative to the more commonly used retroviral vectors in human gene therapy. Specifically, the adeno-associated virus 2 (AAV), a human parvovirus, has gained particular attention in view of its nonpathogenic nature as well as its remarkable site-specificity of integration into the human chromosome. Using the recombinant AAV vector system, it is feasible to obtain high-efficiency transduction of slow- or non-cycling primary hematopoietic stem and progenitor cells, without the need for prestimulation with cytokines, which could potentially lead to differentiation of these cells before transplantation.