Interindividual variability in transgene mRNA and protein production following adeno-associated virus gene therapy for hemophilia A.

An increasing trend in the percentage of hepatocytes that were stained positive for vector genomes was observed across the three dose levels: 1.3% (6 × 10 12 vg per kg body weight), 32% (4 × 10 13 vg per kg body weight) and 45–52% (6 × 10 13 vg per kg body weight), with most positive nuclei containing multiple foci (Fig. 1b,c ).

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