DNA damage/p53, innate immune, and unfolded protein responses are activated in primate liver after toxic, high-dose AAV-SMN1 delivery.

Mol Ther Adv · 2026 · PMC13148890 · PMID 42137263

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18 Although UPR activation is highly significant and dose-dependent, liver toxicity is likely driven by multiple converging mechanisms, including the pro-apoptotic UPR itself, the DNA damage/p53 response, and rapid innate immune activation triggered in NHP livers after high-dose AAV-SMN1 administration.

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