Drug intelligence / Profile preview

AAV8-mF8

Development stage
Preclinical
Lead developer
Queen's University
Modality
Gene Therapies
Administration
Intravenous
01

Overview

AAV8-mF8 is a recombinant adeno-associated virus serotype 8 (AAV8) gene therapy vector encoding murine coagulation factor VIII (mF8). Developed at Queen's University, it serves as a preclinical research tool to evaluate gene therapy strategies for hemophilia A in mouse models, such as F8 knock-out (F8KO) mice. The vector is designed to deliver a codon-optimized mF8 transgene to hepatocytes, enabling the expression and secretion of functional murine factor VIII to restore hemostasis. In research settings, AAV8-mF8 has been utilized to investigate the transcriptional efficiency, durability, and potential of various regulatory elements, including native F8 enhancers and liver-specific promoters, to overcome the decline in transgenic expression typically observed after vector delivery.

Other names
AAV8-mF8 vectorAAV-8-mF8 vectorAAV 8-mF8 vectorAAV8-mFVIIIAAV-8-mFVIIIAAV 8-mFVIIIAAV8-mFVIII vectorAAV-8-mFVIII vectorAAV 8-mFVIII vector
02

Targets

F8 (Coagulation Factor VIIIa)

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