Figure 1.
Regional variation of AAV-cFVIII seen in the liver after long-term follow-up in the hemophilia A dog model, quantified using drop-phase ddPCR. NR, dogs with plasma FVIII:C levels that remained below the limit of detection but showed evidence of improved whole blood clot times compared with untreated hemophilia A dogs.2 (A) AAV-cFVIII VG copies quantified in copies per diploid genome. (B) cFVIII mRNA expression normalized to beta-2-microglobulin (B2M). NR, nonresponder; vgDNA, vector genome DNA.

Regional variation of AAV-cFVIII seen in the liver after long-term follow-up in the hemophilia A dog model, quantified using drop-phase ddPCR. NR, dogs with plasma FVIII:C levels that remained below the limit of detection but showed evidence of improved whole blood clot times compared with untreated hemophilia A dogs.2 (A) AAV-cFVIII VG copies quantified in copies per diploid genome. (B) cFVIII mRNA expression normalized to beta-2-microglobulin (B2M). NR, nonresponder; vgDNA, vector genome DNA.

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