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Volume 20, Issue 10, Pages 1863-1870 (October 2012)
Balloon Catheter Delivery of Helper-dependent Adenoviral Vector Results in Sustained, Therapeutic hFIX Expression in Rhesus Macaques Nicola Brunetti-Pierri, Aimee Liou, Priti Patel, Donna Palmer, Nathan Grove, Milton Finegold, Pasquale Piccolo, Elizabeth Donnachie, Karen Rice, Arthur Beaudet, Charles Mullins, Philip Ng Molecular Therapy Volume 20, Issue 10, Pages (October 2012) DOI: /mt Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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Figure 1 Plasma hFIX levels in rhesus macaques. Vector was administrated on day 0. Normal; >30% of normal. Mild; >5–30% of normal. Moderate; ≥1–5% of normal. Severe; <1% of normal. Normal hFIX level is ~5,000 ng/ml. Molecular Therapy , DOI: ( /mt ) Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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Figure 2 Serum ALT and AST levels in rhesus macaques. Normal range for ALT is 5–61 U/l. Normal range for AST is 12–63 U/l. Grade 1 (mild) = 1.1–2.5× upper limit of normal (ULN). Grade 2 (moderate) = 2.6–5.0× ULN. Grade 3 (severe) = 5.1–10× ULN. Grade 4 (potentially life-threatening) >10× ULN. ALT, alanine transaminase; AST, aspartate transaminase. Molecular Therapy , DOI: ( /mt ) Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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Figure 3 Levels of total anti-hFIX antibodies and inhibitors in rhesus macaques. Vector was administered on day 0. BU, Bethesda units. Molecular Therapy , DOI: ( /mt ) Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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Figure 4 Plasma hFIX levels in mice. Mice injected with two different preparations (lot no or lot no ) of HDΔ21.7E4gE-hFIX at 1 × 1012 virus particle/kg. N = 5 per group. Mean ± SD shown. Molecular Therapy , DOI: ( /mt ) Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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Figure 5 Vector biodistribution in rhesus macaques. Vector genome copies were determined by real-time PCR in different organs in rhesus 17664, 17633, and Molecular Therapy , DOI: ( /mt ) Copyright © 2012 The American Society of Gene & Cell Therapy Terms and Conditions
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