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Innate immune response to AAV-based gene therapy vectors: Mechanisms of complement activation and cytokine release

12 Aug 2025

Abstract excerpt

gene delivery. However, the immune response to rAAV presents a major limitation, leading to serious adverse events in clinical trials. This study investigates the interaction between rAAV and the innate immune system. A whole blood assay (WBA) was used to assess complement activation and cytokine release upon stimulation with rAAV in 20 healthy blood donors. Results demonstrate that AAV2 and AAV8 capsids can...

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PMID: 40917687
DOI: 10.1016/j.omtm.2025.101551

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