Drug intelligence / Profile preview

AAV8-TBG-GFP-mESR1-shRNAmir

Development stage
Preclinical
Lead developer
Universidade de São Paulo
Modality
Viral-delivered RNAi → In Vivo RNAi → Gene Silencing → Gene Therapies
Administration
Intravenous
01

Overview

AAV8-TBG-GFP-mESR1-shRNAmir is a hepatocyte-specific gene knockdown vector delivered via an adeno-associated virus serotype 8 (AAV8) capsid. It utilizes the thyroxine-binding globulin (TBG) promoter to drive the expression of a microRNA-adapted short hairpin RNA (shRNAmir) targeting the mouse Estrogen Receptor 1 (Esr1) gene, which encodes Estrogen Receptor Alpha (ERα). The construct also includes a Green Fluorescent Protein (GFP) reporter for visualization of transduction. This tool is primarily used in preclinical research to investigate the role of hepatic ERα signaling in metabolic disorders such as Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD). Studies have shown that hepatocyte-specific knockdown of ERα using this vector exacerbates high-fat diet-induced steatosis, liver injury, and glucose intolerance in female mice.

02

Targets

ESR1 (ERα)

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