Systemic administration of the lentiviral vector in ADA−/− and
ADA+/− mice. (a) Kaplan–Meier survival curves of
ADA−/− mice when treated with
5.0 × 1010 TU/kg (N = 8),
2.5 × 1010 TU/kg (N = 6), or
untreated (N = 4). (b) Box and whisker (10–90th
percentile) plots of tissue vector copy number from ADA−/− mice
at 6 months (N = 2 for each dose). (c) Box and whisker
(10–90th percentile) plots of ADA enzyme activity at 6 months
(N = 2 for each dose). (d). Floating bar (min–max)
plots of tissue vector copy number over time (4 months, N = 5;
6 months, N = 2; 12 months, N = 1) in
ADA−/− mice treated with
5.0 × 1010 TU/kg. (e) Box and
whisker (10–90th percentile) plots of tissue vector copy number from
ADA−/− mice (N = 4) and ADA+/− littermates
(N = 6) at 6 months (combined data for 2.5 and
5.0 × 1010 TU/kg dose).