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MCU siRNA

Development stage: Preclinical
Lead developer: University of Colorado
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: Parenteral

Overview

MCU siRNA is a small interfering RNA designed to silence the expression of the Mitochondrial Calcium Uniporter (MCU), a protein complex responsible for the uptake of calcium into the mitochondria. In the context of acute myeloid leukemia (AML), research has shown that venetoclax-resistant leukemia stem cells (LSCs) upregulate calcium signaling and rely on high mitochondrial calcium levels to drive oxidative phosphorylation (OXPHOS). By targeting MCU with siRNA, researchers can reduce mitochondrial calcium levels, suppress calcium-dependent TCA cycle activity, and impair the survival and function of these resistant LSCs. This approach is being explored as a therapeutic strategy to overcome venetoclax resistance in AML.

Other names: mitochondrial calcium uniporter siRNA

Targets

MCU (Mitochondrial calcium uniporter)

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