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HMGCR siRNA
- Development stage
- Preclinical
- Lead developer
- University of Pittsburgh
- 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
- Administration
- Intravenous, Subcutaneous
01
Overview
HMGCR siRNA refers to small interfering RNA molecules designed to silence the expression of 3-hydroxy-3-methyl-glutaryl-coenzyme A reductase (HMGCR), the rate-limiting enzyme in the mevalonate pathway for cholesterol biosynthesis. In the context of acute myeloid leukemia (AML), research has demonstrated that chemotherapy agents such as cytarabine and decitabine can upregulate HMGCR expression, which subsequently enhances the secretion of immunosuppressive exosomes by leukemic blasts. HMGCR siRNA has been utilized in preclinical research to show that knocking down HMGCR can block this chemotherapy-induced increase in exosome production. This suggests that targeting the HMGCR pathway via RNA interference could potentially mitigate exosome-mediated chemoresistance and improve anti-leukemia immunity in AML patients.
- Other names
- siRNA targeting HMGCRHMG-CoA reductase siRNA
02
Targets
HMGCR (3-hydroxy-3-methylglutaryl-coenzyme A reductase)
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