AAV9/hMECP2(v2) Provides Tighter Regulation of Transgene Expression
In Mecp2−/y mice, the second-generation vector genome generates fewer cDNA copies per viral genome in specific organs. n = 4 to 5 mice per treatment. **p ≤ 0.004 for heart, liver, and lumbar spinal cord; *p = 0.04 for cervical spinal cord; ##p = 0.001 for liver; #p ≤ 0.05 for heart, cervical spinal cord, and thoracic spinal cord. For clarity, p values are only shown for comparisons against mice treated with 1 × 1012 vg AAV9/hMECP2(v1). Data points are mean ± SEM. CC, cervical spinal cord; LC, lumbar spinal cord; TC, thoracic spinal cord.