phFIX vector

The phFIX vector refers to a plasmid-based gene therapy delivery system designed to express human Coagulation Factor IX (FIX) for the treatment of Hemophilia B. In early gene therapy research and clinical trials, phFIX was often utilized as a non-viral delivery method, where the plasmid DNA encoding the human FIX gene was administered directly (e.g., via intramuscular injection or hydrodynamic limb vein delivery) to induce local or systemic production of the clotting factor. While naked plasmid DNA delivery generally resulted in low and transient expression levels compared to modern viral vectors, the phFIX vector served as a foundational tool in establishing the feasibility of gene-based replacement therapy for clotting disorders. The mechanism involves the uptake of the plasmid by host cells, followed by the transcription and translation of the human FIX protein, which is then secreted into the circulation to restore hemostasis.

Other names

plasmid human Factor IXpCMV-hFIXpAAV-hFIX