Fig. 2: Levels of vector DNA and transgene protein. | Gene Therapy

Fig. 2: Levels of vector DNA and transgene protein.

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

Fig. 2

For the 22 F0 males who received AAV5-hFVIII-SQ and sired offspring after mating at 4 or 37 days post-dose, vector genome copy number in individual tissue samples from (A) liver or (B) testes, and (C) hFVIII-SQ protein plasma levels. Data are shown for the 22 F0 males who received AAV5-hFVIII-SQ and sired offspring and their 139 F1 male and female pups. No vector genome DNA was detected in any F0 male who received a vehicle injection. F0 males mated at 4 days post-dose were not evaluated for hFVIII protein presence. For the hFVIII protein assay, the LLOQ was 2.35 ng/mL; samples with protein levels below the LLOQ are shown as 0 ng/mL. hFVIII human Factor VIII, LLOQ lower limit of quantitation.

Back to article page