Shows sustained human FVII expression in mice, suggesting potential for FVII deficiency treatment.
genome copies (gc)/kg, we observed 48 weeks of functional hFVII expression which peaked at 16 IU/mL and stabilized at 7 IU/mL. These results support the pre-clinical development of AAV8-mediated delivery employing the splice-variant hFVII(-22) for patients suffering from FVII deficiency.
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D’Amico et al. (2025) studied this question.
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