Figure 1.

Systemic transduction after intravenous injection of sc- and ssAAV9-GFP into fetal and neonatal mice. The ssAAV9 packaged a CMV promoter driving GFP expression with a downstream WPRE sequence. A) This expression cassette was flanked by the viral inverted terminal repeats (ITRs). B) The scAAV9 configuration also packaged the CMV-GFP expression cassette but without the WPRE sequence. The vectors were intravenously administered to fetal mice in utero via the vitelline vessels or to neonatal mice via the superficial temporal vein. One month after injection, organs were harvested and observed by stereoscopic fluorescence microscope. ssAAV9-injected fetal heart (C), neonatal liver (D), neonatal kidney (E), fetal bones in the paw (F) and femur (G), neonatal vertebrae and ribs (H), fetal pulmonary vein (I), fetal skeletal muscle (J), neonatal penis (K), and neonatal testicle (L). In the same frames, scAAV9-injected and negative control neonatal diaphragm muscle (M), fetal heart (N), and neonatal pancreas (O).