Drug intelligence / Profile preview

CTR1siRNA

Development stage
Preclinical
Lead developer
Augusta 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

CTR1siRNA is a small interfering RNA (siRNA) designed to silence the expression of the high affinity copper uptake protein 1 (CTR1), also known as SLC31A1. CTR1 is the primary transporter responsible for the influx of dietary copper into cells. In preclinical cardiovascular research, CTR1 has been identified as a mechanosensor in endothelial cells that responds to disturbed blood flow by promoting excessive copper uptake. This accumulation of copper leads to cuproptosis, a form of regulated cell death characterized by mitochondrial protein aggregation and respiratory dysfunction, which contributes to the development of atherosclerosis. By knocking down CTR1 expression, this siRNA prevents copper-mediated mitochondrial damage and has demonstrated the ability to reduce atherosclerotic lesions in animal models.

Other names
CTR1 siRNACTR-1 siRNACTR 1 siRNAsiRNA targeting CTR1
02

Targets

SLC31A1 (Human copper transporter 1)

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