Figure 6.

ADP-LNPs containing Cas9 mRNA/gRNA can efficiently edit the Ube3a-ats gene and can potentially treat Angelman syndrome. (A) Schematic diagram describing the editing strategy used to upregulate the expression of Ube3a. Cas9/sgRNA/LNP complexes target the paternal allele of Ube3a-ats, which silences Ube3a. Successful editing downregulates the Ube3a-ats expression, allowing the paternal Ube3a allele to be expressed. (B) The bar graph depicts the indel frequencies observed at 10 days postinjection of ADP-LNPs, demonstrating efficient editing (data are represented as mean ± SEM, n = 3 for sham and n = 4 for LNP/Cas9/gRNA treated (***p < 0.001)). (C) Relative expression levels of Ube3a-ats in sham and treated groups, showing a significant reduction in the treated group (*p < 0.05). Data are represented as mean ± SEM, n = 3 for sham and n = 6 for ADP-LNP/Cas9/gRNA treated.