Figure 3.

Comparison of splicing correction by self-complementary adenovirus-associated virus (scAAV) vectors with different numbers of U7-ESE-B cassettes. HeLa S2 cells were transduced with AAV serotype 6 particles of scAAV 5xsU7 and scAAV 1xsU7 (a,b) or scAAV 5xsU7 and scAAV 4xsU7 (c). SMN2 minigene splicing assays a,c and measurements of U7-ESE-B RNA b were performed as in Figure 2. All samples were collected 24 hours post-transduction. The vector concentrations are indicated as vector genome excess over cells. For example, 5× indicates a concentration of 5 vg/cell. Error bars indicate standard deviations from two biological replicates carried out in duplicate.