Table 3

Analysis of T cell responses performed by IFN-γ ELISpot after initial injection (bold) and after readministration. The time points for study are described in table S1. Most of the samples tested for T cell responses to the AAV capsid or the RPE65 transgene product were negative throughout the initial (18) and FO studies. A few samples tested positive in the assay (for example, CH12, FO week 6); however, these samples were negative the following week, suggesting either that the positive readings were false positives or that there was weak or transient T cell activation. Thus, there were no cell-mediated T cell responses detectable in peripheral blood, a result in agreement with the lack of local inflammation. Pos, positive (>50 SFUs per million cells plated) and at least threefold the medium-only control; Neg, negative (<50 SFUs per million cells plated) or less than threefold the medium-only control; Bkg, high background/not interpretable (medium control >100 SFUs per million cells plated).

SubjectAntigend0/FOd0FO
week 1
Week 2/FO
week 2
FO
week 3
Week 4/FO
week 4
FO
week 5
FO
week 6
FO
week 7
FO
week 8
d90/FOd90
CH12AAVNeg/NegNegNeg/NegNegNeg/NegNegNegNegNegNeg*/Neg
RPE65Neg/NegNegNeg/NegNegNeg/NegNegNegNegNegNeg*/Neg
CH11AAVNeg/BkgBkgNeg/BkgBkgNeg/BkgBkgPosNegNegNeg/Neg
RPE65Neg/BkgBkgNeg/BkgBkgNeg/BkgBkgPosNegNegNeg/Neg
NP01AAVNeg/NegNegNeg/BkgNegNeg/BkgNegNegBkgNegNeg/Bkg
RPE65Neg/NegNegNeg/BkgNegNeg/BkgPosNegBkgNegNeg/Bkg

*Poor viability of cells.

†Positive result likely due to high background reactivity.