Figure 2.
Time-course of vector-mediated AAT expression and enzyme-linked immunosorbent spot (ELISPOT) responses to AAV1 capsid peptides in patients receiving (A) 2.2 × 1013 or (B) 6.0 × 1013 vgs intramuscularly of a recombinant AAV1 vector expressing the M-AAT gene. Serum M-specific AAT levels are plotted at the top of each panel and interferon-gamma ELISPOT responses to an AAV1 capsid peptide library are plotted at the bottom of each panel. M-AAT levels past day 90 for subjects 201 and 303 are not shown as they resumed protein replacement at this time-point. The day 365 sample for subject 202 could not be tested because of severe hemolysis. ELISPOT responses are characterized as – (negative in both ex vivo and cultured assays),±(positive in cultured assay but negative in ex vivo assay) or + (positive in both ex vivo and cultured assays). Samples for ELISPOT analysis were not available beyond day 90 for subjects 202, 203 and 302. Reprinted with permission from Brantly et al. (15).