Extended Data Fig. 7: Immunogenicity assessment of AAV5-CRISPR/Cas9-based in vivo genome editing in NHPs.
From: Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
a,b, Antibodies against AAV5 capsid protein (a) and Cas9 protein (b) were measured in sera from the study animals using a Luminex bead-based assay. Results are presented as concentration of IgG against AAV5 and Cas9 in each individual animal’s serum at each time point. All samples were run in triplicate. c,d, ELISpots were performed to measure Cas9-specific CD8 T-cell responses (c) and Cas9-specific CD4 T-cell responses (d). Peripheral blood mononuclear cells from individual animals were stimulated with Cas9 peptide pools and assayed for IFN-γ production. Data are presented as the number of IFN-γ spot-forming cells per million cells.
