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miR-101a
- Development stage
- Preclinical
- Lead developer
- Children's Cancer Institute
- Modality
- RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intratumoral
01
Overview
miR-101a is a microRNA that acts as a tumor suppressor, particularly in the context of Mixed-Lineage Leukemia (MLL)-mediated acute myeloid leukemia (AML). It is frequently downregulated in leukemic stem cells (LSCs). miR-101a functions by targeting and suppressing the expression of the epigenetic regulator Enhancer of zeste homolog 2 (EZH2), as well as pro-survival proteins such as Myeloid Cell Leukemia 1 (MCL-1) and B-cell lymphoma 2 (BCL-2). Forced expression of miR-101a has been shown to impair LSC proliferation, induce apoptosis, and delay leukemia development in preclinical models, suggesting its potential as a therapeutic strategy to eradicate drug-resistant LSCs.
- Other names
- microRNA-101amicroRNA101amicroRNA 101a
02
Targets
MCL1 (Myeloid cell leukemia sequence 1)Proto-oncogene c-Fos messenger RNASTMN1 (Stathmin-1)EZH2 (Histone-lysine N-methyltransferase EZH2)
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