Figure 1.

Schematic of ABCA4 dual AAV vector platforms created for this study. (A) Full-length ABCA4 expression cassette. (B) Overlap vector showing the three overlaps tested (B1–B3). (C) Transplicing vector. (D) Hybrid vector platform. Promoter, cytomegalovirus immediate early/chicken β-actin chimeric promoter; ITR, inverted terminal repeat; polyA, polyadenylation signal; splice donor/acceptor sites marked black regions; AP overlap sequences marked as hatched regions. AAV, adeno-associated virus; ABCA4, ATP binding cassette transporter A4; AP, alkaline phosphatase.