Extended Data Fig. 4: Detection of hFVIII-SQ RNA transcript in patient liver biopsy samples. | Nature Medicine

Extended Data Fig. 4: Detection of hFVIII-SQ RNA transcript in patient liver biopsy samples.

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

Extended Data Fig. 4

hFVIII-SQ RNA levels detected in patient liver samples normalized to endogenous reference RNAs a) beta-actin, b) Rplp0, and c) YWHAZ. d) Correlation of RNA quantified by ddPCR with total RISH area signal in one tissue section per participant; RISH foci sum normalized to tissue area, μm2 (Pearson correlation coefficient, r = 0.90; p = 0.03, two-tailed). e) Correlation between plasma FVIII activity and liver hFVIII-SQ RNA; Participants 1 and 15 had no or barely detectable plasma FVIII activity (black diamonds). CS, chromogenic substrate assay; hFVIII-SQ, B domain-deleted human factor VIII; P, participant; RISH, RNA in situ hybridization; Rplp0, ribosomal protein lateral stalk subunit P0; YWHAZ, tyrosine 3-monooxygenase/tryptophan 5-monooxygenase activation protein zeta.

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