FIGURE 1.

Map and structure of MECP2 gene, protein and editing vector. (A) Genomic structure and protein domains of MECP2. MeCP2 protein domains and their corresponding locations in Exon 3 and 4 are color coded. (B) Map of the single-stranded AAVHSC-226 editing vector genome. The editing vector was designed to correct mutations in MECP2 Exons 3 and 4 and two unique linker sequences L1 and L2 were included in Introns 2 and 3, respectively. The editing vector was also designed to insert a promoterless T2A-Venus ORF cassette immediately downstream of Exon 4.