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AIF-1 shRNA
- Development stage
- Preclinical
- Lead developer
- Beijing Chaoyang Hospital
- Modality
- Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies, RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
AIF-1 shRNA is a short hairpin RNA therapeutic candidate designed to silence the expression of Allograft Inflammatory Factor-1 (AIF-1). AIF-1 is a protein predominantly enriched in granulocyte/monocyte-like leukemic subpopulations and is highly expressed in acute monocytic leukemia (AMoL) and acute myelomonocytic leukemia (AMML). By knocking down AIF-1, the drug inhibits the self-renewal, migration, and infiltrative capacity of leukemic cells. Mechanistically, AIF-1 knockdown leads to the downregulation of C-C chemokine receptor type 2 (CCR2) and the MAPK signaling pathway, which are critical for extramedullary infiltration (EMI) in AML. Preclinical studies in THP-1 cell-derived xenograft models have shown that AIF-1 knockdown suppresses liver infiltration and prolongs survival.
- Other names
- AIF-1 knockdownAIF1 knockdownAIF 1 knockdownAIF1 shRNAAIF-1 shRNAAIF 1 shRNA
02
Targets
AIF1 (Allograft inflammatory factor 1)
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