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epsin siRNA
- Development stage
- Preclinical
- Lead developer
- Boston Children's Hospital
- Modality
- Chemically Modified siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Nanoparticles → Drug Delivery Systems, Conjugated siRNA → Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intravenous
01
Overview
Epsin siRNA is an experimental therapeutic consisting of nanoparticle-encapsulated small interfering RNAs (siRNAs) designed to silence the expression of epsins 1 and 2, specifically within the liver. Epsins are a family of ubiquitin-binding endocytic adaptors that play a critical role in the regulation of the low-density lipoprotein receptor (LDLR). Under atherogenic conditions, Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) binds to LDLR and utilizes epsins to facilitate the receptor's internalization and subsequent lysosomal degradation. By depleting liver epsins, this siRNA treatment prevents the PCSK9-mediated degradation of LDLR, thereby increasing the availability of the receptor on the surface of hepatocytes. This mechanism enhances the clearance of low-density lipoprotein cholesterol (LDL-C) from the bloodstream, effectively reducing dyslipidemia and inhibiting the progression of atherosclerosis.
- Other names
- nanoparticle-encapsulated siRNAs targeting epsins 1 and 2epsin 1/2 siRNA
02
Targets
EPN1 (Epsin 1)
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