No evidence for germ-line transmission following prenatal and early postnatal AAV-mediated gene delivery

Background: Recombinant adeno-associated viruses have been used successfully in a number of pre-clinical and clinical gene therapy studies. Since there is a broad consensus that gene therapy must not lead to germ-line transmission, the potential of such vectors for inadvertent gene transfer into germ cells deserves special attention. This applies in particular to pre- or perinatal vector application which has been considered for diseases presenting with morbidity already at birth. Methods: AAV serotype 2-derived vectors carrying a beta-galactosidase reporter gene or human clotting factor IX cDNA were injected in…