Figure 6.

Comparison of transduction profiles of parental adeno-associated virus serotype 9 (AAV9) and variants in heart and liver. (a) Heart-to-liver ratios for luciferase expression levels and (b) vector genome copy numbers were derived from average values. Variant 9.45 (white bars) demonstrates high heart-to-liver expression and vg ratios and correspondingly high liver-detargeting efficiency. Variant 9.68 displays heart-to-liver ratios lower than parental AAV9 vectors and hence preferential liver transduction. (c) Fluorescence micrographs of heart, liver and skeletal muscle (gastrocnemius) tissue sections from mice injected with AAV9 or AAV9.45 vectors packaging the CBA-tdTomato cassette. Images were obtained at ×20 (liver) or ×10 (heart and skeletal muscle) magnification using an Olympus microscope equipped with a Hamamatsu digital camera. Tissue sections from untreated mice are shown as control. All experiments were carried out in duplicate.