Fig. 6. Vector is detected in in utero rAAV1-CMV-hGAA-transduced
diaphragms by QC-PCR. Quantitative-competitive PCR was performed to determine
vector genome copies in in utero vector-treated and untreated diaphragm
samples that were previously analyzed in
Fig. 4. PCR was performed after
mixing increasing amounts of competitor DNA (100 to 108
copies) and 200 ng of total DNA isolated from the diaphragms of
Gaa-/- untreated mice or Gaa-/- mice
in utero-treated with 8.14x1010 particles of
rAAV1-CMV-hGAA. Representative samples (3 of the 8 assayed) are
listed, with their respective values representing estimated vector copies per
diploid genome and average GAA activity. Raw QC-PCR data from each diaphragm
is shown on the far right.