American Society of Hematology

Fig. 6.

$Fig. 6. Detection of rAAV CAGG-FIX transgene in extrahepatic tissue. / Genomic DNA was extracted from liver, kidney, spleen, and testis tissue from one macaque (monkey 3) 9 months after liver-targeted delivery of rAAV in 2 fractionated doses and from the liver of a naive macaque (Con). Then, 1 μg genomic DNA was used for PCR amplification employing transgene-specific primers designed to amplify a 521-bp product. Proviral copy number was deduced from standards that consisted of serial dilutions of vector DNA (5 × 10−3 to 5 copies) in 1 μg control genomic DNA. The integrity of DNA was determined by amplifying a 604-bp region of the rhesus β-gene and is shown at the bottom of the panel.$

Detection of rAAV CAGG-FIX transgene in extrahepatic tissue.

Genomic DNA was extracted from liver, kidney, spleen, and testis tissue from one macaque (monkey 3) 9 months after liver-targeted delivery of rAAV in 2 fractionated doses and from the liver of a naive macaque (Con). Then, 1 μg genomic DNA was used for PCR amplification employing transgene-specific primers designed to amplify a 521-bp product. Proviral copy number was deduced from standards that consisted of serial dilutions of vector DNA (5 × 10⁻³ to 5 copies) in 1 μg control genomic DNA. The integrity of DNA was determined by amplifying a 604-bp region of the rhesus β-gene and is shown at the bottom of the panel.

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