Fig. 1: Study design schematic. | Gene Therapy

Fig. 1: Study design schematic.

From: Lack of germline transmission in male mice following a single intravenous administration of AAV5-hFVIII-SQ gene therapy

Fig. 1

F0 male mice received an injection of either vehicle or 6 × 1013 vg/kg AAV5-hFVIII-SQ and were mated afterward at two different timepoints to naïve females. Some F0 males were mated 4 days afterward, when transgene DNA in semen was estimated to be at peak concentration, and some were mated 37 days after injection, when a full spermatogenesis cycle was expected to complete. F0 males were assessed for transgene DNA in the testes and liver and for hFVIII-SQ protein in plasma. F1 pups were assessed for transgene DNA in the liver. hFVIII human Factor VIII.

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