Correlation between In Vitro and In Vivo AAV NAb Assays
(A) Experimental workflow of the in vivo NAb assay (see Materials and Methods for assay details). (B and C) Dot plot of serum samples tested by both the in vitro and in vivo assays. Each dot represents the serum sample from an individual large animal. CM, Cynomolgus macaque; MM, marmoset; SH, sheep. Red dots, p < 0.05 compared against the naive mouse serum controls in the in vivo assay (normalized to 100% serum hA1AT; Student’s t test). Note that the majority of sero-positive (>1:5 by in vitro assay) sera of CM showed in vivo inhibition (B), but not the sero-positive sera of MM and SH (C).