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siRNA-RMRP
- Development stage
- Preclinical
- Lead developer
- Peking University
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
siRNA-RMRP is a small interfering RNA (siRNA) designed to target and silence the long non-coding RNA (lncRNA) known as RMRP (RNA Component of Mitochondrial RNA Processing Endoribonuclease). In the context of relapsed and refractory acute myeloid leukemia (R/R AML), RMRP is significantly enriched in the mitochondria where it regulates oxidative phosphorylation, mitochondrial genome transcription, and epigenetics. By knocking down RMRP expression, siRNA-RMRP attenuates mitochondrial metabolic capacity, reduces mtDNA copy number, and induces mitophagy through pathways such as BNIP3L/Nix and PINK1/Parkin. This targeted depletion of RMRP has been shown to increase the sensitivity of AML cells to chemotherapy agents like Cytarabine and Doxorubicin, suggesting its potential as a therapeutic strategy to overcome chemoresistance.
- Other names
- RMRP siRNAsi-RMRP
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