It is recommended to set early (1-2 weeks, confirm the onset of effect), primary endpoint (3-6 weeks, compare expression and distribution) and sustained endpoint (8-12 weeks or longer). Detection should include at least two layers of vector DNA, transgenic mRNA, and protein/functional readout; for tissue distribution issues, non-target tissue and empty vector/negative controls should be added. For actively proliferating tissues, long-term expression decline may result from vector genome dilution rather than batch quality issues. [12-2,12-3]
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5. Reference Doses and Detection Time Points for AAV Studies
AAV, Administration Routes, Dosing, and Expression Assessment
Updated Oct 19, 2026