FIGURE 4.
Targeting the PTM closer to the intended 3′ splice site results in a substantial increase in trans-splicing efficiency. The panel above the histogram shows a schematic of two different PTMs and the location of their binding domain with respect to the target acceptor site. The binding domain of CF-PTM53 is located ~400 bases downstream of CF-PTM50. Both PTMs have a spacer sequence that is shorter than all previous constructs by 50 bases. Values are mean ± standard error of three separate experiments. (BD) Binding domain.
