AAV8 Serotype
Highly efficient liver-tropic serotype widely used in clinical hepatic gene therapy. Discovered from rhesus macaque tissue. Superior liver transduction versus AAV2.
Characteristics
10-100 fold higher liver transduction than AAV2. Fast onset of transgene expression (1-2 weeks). Moderate pre-existing immunity (~40% seropositive). Uses laminin receptor (LamR) for hepatocyte binding. Efficient hepatocyte transduction at low doses. Good safety profile in clinical trials.
Applications
Hemophilia A and B gene therapy (multiple clinical trials). Liver-directed metabolic disorders. Ornithine transcarbamylase deficiency. Glycogen storage diseases. Acute intermittent porphyria. Most common serotype for liver gene therapy trials.
Literature References
- Nathwani et al. (2011). Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. N Engl J Med - Nathwani 2011 Hemophilia B
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