Figure 1.

Outcomes following AAV5-hFVIII-SQ treatment in neonates (2 days old) versus adults (8 weeks old).

(A) Plasma hFVIII-SQ levels at 8 weeks post-treatment with AAV5-hFVIII-SQ (3.5 × 10¹³ vg/kg) in neonates versus adults. (B) hFVIII-SQ protein concentrations in plasma by time point in neonates and adults treated with AAV5-hFVIII-SQ (8.9 × 10¹¹ vg/mouse). (C) Total AAV5-hFVIII-SQ vector genomes in liver by time point in neonates and adults treated with AAV5-hFVIII-SQ (8.9 × 10¹¹ vg/mouse) detected by qPCR. (D) Total hFVIII-SQ RNA in liver by time point in neonates and adults treated with AAV5-hFVIII-SQ (8.9 × 10¹¹ vg/mouse) detected by RT-qPCR. Data are shown as mean ± standard deviation in red for mice dosed as neonates and blue for mice dosed as adults. AAV5-hFVIII-SQ, adeno-associated virus serotype 5 vector encoding a B-domain-deleted human FVIII; FVIII, factor VIII; hr, hours; PCR, polymerase chain reaction; qPCR, quantitative real-time PCR; RT-qPCR, reverse transcription followed by quantitative real-time PCR; vg, vector genome; wk, weeks.