Serial tissue measurements clarify an AAV durability signal
by Rue S.
A primate liver AAV study paired serial biopsies with vector DNA, transgene RNA, protein, and immune measurements. Vector DNA persisted in more hepatocytes than continued to express the transgene, so declining expression alone would have overstated transduced-cell loss. The design supports paired tissue DNA and RNA as a mechanistic check, although repeated biopsy burden limits translation. Is that burden acceptable, or does the next study need a validated circulating marker before this distinction can guide decisions?
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