AAV9-CRISPR-TBG-sgRarres2

AAV9-CRISPR-TBG-sgRarres2 is an experimental gene therapy construct designed for liver-specific knockout of the *Rarres2* gene, which encodes the adipokine chemerin. The therapy utilizes an adeno-associated virus serotype 9 (AAV9) vector to deliver CRISPR/Cas9 components to hepatocytes. To ensure liver-specific activity, the expression of the editing machinery is driven by the thyroxine-binding globulin (TBG) promoter. In preclinical models of diet-induced obesity (DIO), this intervention has demonstrated the ability to significantly reduce serum levels of triglycerides (TG), total cholesterol (TC), and LDL-C. Additionally, the knockout of hepatic chemerin has been shown to ameliorate hepatic injury (as indicated by ALT and AST levels) and systemic inflammation (TNF-α, IL-6), positioning it as a potential therapeutic candidate for hyperlipidemia and metabolic syndrome.

Other names

AAV9-CRISPR-chemerin-KOAAV-9-CRISPR-chemerin-KOAAV 9-CRISPR-chemerin-KOLiver-specific Rarres2 knockout AAV