Figure 2.

Development of GenX AAV vectors. The D sequence at the 3′ end in the viral inverted terminal repeat (ITR) contains the binding site for a cellular protein, FKBP52, phosphorylated forms of which strongly inhibit viral second-strand DNA synthesis. The D sequence at the 5′ end in the ITR contains the binding site for NF-κB-repressing factor, a negative regulator of transcription. Removal of these sequences led to the generation of GenX AAV vectors that circumvent these problems, and led to more efficient transgene expression.