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CYP7A1 siRNA
- Development stage
- Preclinical
- Lead developer
- Saint Louis 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
- Subcutaneous
01
Overview
CYP7A1 siRNA is a small interfering RNA (siRNA) designed to target and silence the expression of cytochrome P450 family 7 subfamily A member 1 (CYP7A1), the rate-limiting enzyme in primary bile acid synthesis. By knocking down CYP7A1 expression, this RNA interference (RNAi) therapy aims to reduce the synthesis of primary bile acids, thereby mitigating hepatocellular injury, cholestasis, and progressive fibrosis. It is being investigated as a potential therapeutic strategy for cholestatic liver diseases, such as biliary atresia (BA) and primary sclerosing cholangitis (PSC).
- Other names
- CYP7A1 RNAi agentCYP-7A1 RNAi agentCYP 7A1 RNAi agentCYP7A1 small interfering RNACYP-7A1 small interfering RNACYP 7A1 small interfering RNACyp7a1 siRNACyp-7a1 siRNACyp 7a1 siRNA
02
Targets
CYP7A1 (Cholesterol 7alpha-hydroxylase)
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