Anti-AAV9 IgG responses are sustained and cross-reactive to distinct AAV capsid serotypes
(A) Dendrogram showing the phylogenetic relatedness of AAV VP1 amino acid sequences from native capsid serotypes 1–13 and the engineered capsid LK03. (B) Anti-AAV IgG endpoint titers to AAV capsids 1, 2, 5, 8, 9, and LK03 in treated infants were measured using ELISA. The x axis indicates day of serum collection post-AAV infusion. Gray zones indicate levels below the limit of assay sensitivity with a lower sensitivity for pre-infusion samples. Area between dashed black lines indicates the cut-off range to preclude gene therapy treatment, accommodating different levels of assay sensitivity. Pre-infusion sera were not available for patients 1 and 6.