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hydrophila was treated with escalating PMP concentrations (0, 0.1, 1, and 10 mg/mL, designated as Control, PMP1, PMP2 and PMP3), and D-cycloserine (0.5 mg/mL, positive control) (Fig

Pharmacokinetics, Biodistribution, Immunogenicity, and Model-Informed-Based PK/PD Model of a Next-Generation Advanced Novel Gene Therapy for Hemophilia