Figure 7.

2'-MOE-SSO 91 greatly increases amounts of wild-type β-globin mRNA. In these droplet digital polymerase chain reaction analyses, y-axes indicate relative expression of the wild-type (WT) β-globin (HBB) gene normalized to the housekeeping β-actin gene in (A) IVS2-745/b0 heterozygous sample, P4, and (B) IVS2-745 homozygous sample, P5. In both samples, 2'-MOE-SSO scramble and 2'-MOE-SSO 91 treatment were at the dose of 50 mM. All statistical comparisons (ANOVA/Kruskal-Wallis) are tested against scramble-treated and non-treated sample; n=3. *P<0.1, **P<0.01, ****P<0.0001.