Figure 1.
ssAAV9-HexB-P2A-HexA Construct Design
The human HEXA and HEXB genes were synthesized with a self-cleaving linker, P2A, between them and with a CAG promoter upstream to drive expression. The entire construct was designed between the appropriate AAV inverted terminal repeat (ITR) elements, and an SV40 element was included to promote protein expression in mammalian cells.