Figure 3. Varying PTOs in targeting oligos.

All oligos in this figure are complementary to the non-transcribed strand’s first potential start codon TTG, similar to F5-3 in Fig. 1. (a) Oligo toxicity and targeting efficiency as a function of PTO modifications. PTO bond position shown in gray. ‘Total alive cells’ was estimated as the total gated PI- cells in 200 uL from a 24-well plate 48 hrs post oligo transfection. (b) Correction efficiencies of new oligo designs with reduced PTO bonds and using FU modified base. n = 4.