biostudies-literatureUnknown34(4)Ruzicka MRetinoic acid-inducible gene-I (RIG-I) is a cytoplasmic immune receptor sensing viral RNA. It triggers the release of type I interferons (IFN) and proinflammatory cytokines inducing an adaptive cellular immune response. We investigated the therapeutic potential of systemic RIG-I activation by short 5'-triphosphate-modified RNA (ppp-RNA) for the treatment of acute myeloid leukemia (AML) in the syngeneic murine C1498 AML tumor model. ppp-RNA treatment significantly reduced tumor burden, delayed disease onset and led to complete remission including immunological memory formation in a substantial proportion of animals. Therapy-induced tumor rejection was dependent on CD4⁺ and CD8⁺ T cells, but not on NK or B cells, and relied on intact IFN and mitochondrial antiviral signLeukemia1017-1026https://www.ebi.ac.uk/biostudies/studies/S-EPMC7214254biostudies-literatureRIG-I-based immunotherapy enhances survival in preclinical AML models and sensitizes AML cells to checkpoint blockade.PMC7214254Endres SMeinl HBoehmer DFRLichtenegger FSKoenig LMKocheise LKobold SSubklewe MZeitlhofler MRuzicka MVick BSchnurr MAhlfeld JDuewell PJeremias IFormisano SHeuer EMRothenfusser SfalseRIG-I-based immunotherapy enhances survival in preclinical AML models and sensitizes AML cells to checkpoint blockade.Retinoic acid-inducible gene-I (RIG-I) is a cytoplasmic immune receptor sensing viral RNA. It triggers the release of type I interferons (IFN) and proinflammatory cytokines inducing an adaptive cellular immune response. We investigated the therapeutic potential of systemic RIG-I activation by short 5'-triphosphate-modified RNA (ppp-RNA) for the treatment of acute myeloid leukemia (AML) in the syngeneic murine C1498 AML tumor model. ppp-RNA treatment significantly reduced tumor burden, delayed disease onset and led to complete remission including immunological memory formation in a substantial proportion of animals. Therapy-induced tumor rejection was dependent on CD4⁺ and CD8⁺ T cells, but not on NK or B cells, and relied on intact IFN and mitochondrial antiviral sign2020-01-01T00:00:00Z2020 Apr2025-05-29T21:59:46.773Z2025-05-29T21:59:46.773ZS-EPMC72142543174080910.1038/s41375-019-0639-x