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miR-130a inhibitor
- Development stage
- Preclinical
- Lead developer
- Princess Margaret Cancer Centre
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
miR-130a inhibitors are a class of RNA-targeted therapeutics, typically antisense oligonucleotides or antagomirs, designed to neutralize the activity of microRNA-130a. In the context of hematologic malignancies, specifically t(8,21) acute myeloid leukemia (AML), miR-130a is frequently upregulated and acts as a key regulator of leukemic self-renewal and differentiation blocks. By inhibiting miR-130a, these agents restore the expression of critical targets like CBFβ and TBL1XR1, which in turn destabilizes the AML1-ETO (AE) oncofusion complex and promotes myeloid maturation and apoptosis. Preclinical studies have demonstrated that miR-130a knockdown can significantly reduce leukemic engraftment and blast population in patient-derived xenograft models. Beyond oncology, miR-130a is also studied in cardiovascular diseases and fibrosis due to its role in regulating angiogenesis and TGF-beta signaling.
- Other names
- miR-130a antagomirmiR130a antagomirmiR 130a antagomiranti-miR-130aanti-miR130aanti-miR 130amiR-130a knockdownmiR130a knockdownmiR 130a knockdown
02
Targets
AGO2 (Argonaute RISC catalytic component 2)miR-130a-3p (MicroRNA-130a-3p)
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