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siRNA targeting ion transporter T1
- Development stage
- Preclinical
- Lead developer
- University of Duisburg-Essen
- 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 targeting ion transporter T1 is an experimental small interfering RNA (siRNA) therapeutic candidate under preclinical investigation for the treatment of Wilson's disease [3045]. Developed by researchers at the University of Duisburg-Essen and University Hospital Muenster, the therapy aims to mitigate hepatocellular copper overload and associated toxicity [3045]. In preclinical models, including ATP7B-knockout HepG2 cells and primary human hepatocytes, siRNA-mediated silencing of the copper-sensitive ion transporter T1 significantly reduced copper toxicity, enhanced cell proliferation, improved cell and mitochondrial viability, and downregulated copper-regulated metallothioneins [3045]. This approach represents a potential novel therapeutic strategy for copper detoxification in patients with Wilson's disease [3045].
- Other names
- T1 siRNAT-1 siRNAT 1 siRNAsiRNA targeting T1
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