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. 2024 Oct 2;16(11):2882–2917. doi: 10.1038/s44321-024-00148-5

Figure EV5. In vivo safety study in WT mice through ELISpot assay, AAV9/hAP4B1 treatment generated no B cell response to the hAP4B1 peptides.

Figure EV5

Vectors CBh-hAP4B1 and SYN-hAP4B1 were tested, alongside the vector containing the mouse Ap4b1 gene (mAP4B1). Splenocytes that were prepared from WT mice following were assessed through an ELISpot assay for IFN-γ responses to AP4B1 peptides. (A) Representative images of the spot forming detection revealed following the exposure to the negative control (DMSO), the hAP4B1 peptides and the positive control (Concanavalin A). (B) Treated mice did not show any inflammatory response to the peptides. (C) RT-qPCR of total RNA extracted demonstrated elevated hAP4B1 mRNA expression in the brain, liver and spinal cord of treated WT mice with lower expression within the liver. Data are presented as mean ± SEM, n = 3 per group. Data are analysed by a two-way ANOVA with Tukey’s post hoc multiple comparison test. ns = not significant.