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GalNAc-siTmem141
- Development stage
- Preclinical
- Lead developer
- Northeast Ohio Medical 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
- Administration
- Intravenous, Subcutaneous
01
Overview
GalNAc-siTmem141 is a preclinical-stage small interfering RNA (siRNA) therapeutic conjugated to N-acetylgalactosamine (GalNAc) designed to target transmembrane protein 141 (TMEM141) in hepatocytes. Developed by researchers at the University of Arizona College of Medicine – Phoenix and Northeast Ohio Medical University, the drug is designed to treat metabolic dysfunction-associated steatohepatitis (MASH) and MASH-associated fibrosis. TMEM141 is a mitochondrial protein that interacts with mitochondrial complex I to regulate reactive oxygen species (ROS) production. By silencing hepatic TMEM141, GalNAc-siTmem141 reduces ROS production and restores hepatocyte nuclear factor 4α (HNF4α) expression, promoting the reversal of high-fat/cholesterol/fructose (HFCF) diet-induced MASH and fibrosis.
- Other names
- GalNAc-siTMEM141GalNAc-siTMEM-141GalNAc-siTMEM 141siTmem141siTmem-141siTmem 141
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