Drug intelligence / Profile preview

PCSK9 AAV

Development stage
Unknown
Lead developer
Genevoyager
Modality
Gene Addition/Replacement → Gene Therapies, Gene Editing → Gene Therapies, Gene Silencing → Gene Therapies
Administration
Intravenous, Intraperitoneal
01

Overview

PCSK9 AAV refers to an adeno-associated virus (AAV) vector, most commonly of the AAV8 serotype, engineered to deliver and overexpress the Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) gene. In cardiovascular research, this tool is primarily utilized to create mouse models of hyperlipidemia and atherosclerosis. By delivering a gain-of-function mutation of the PCSK9 gene (such as the human D374Y variant) to the liver, the vector induces the rapid degradation of low-density lipoprotein receptors (LDLR), resulting in sustained high levels of circulating LDL cholesterol. This method provides a rapid and cost-effective alternative to traditional germline genetic modifications, such as LDLR knockout mice, for studying lipid metabolism and evaluating potential anti-atherosclerotic therapies.

Other names
PCSK9-expressing adeno-associated virusPCSK-9-expressing adeno-associated virusPCSK 9-expressing adeno-associated virusAAV-mediated PCSK9 expression
02

Targets

LDLR (Low-density lipoprotein receptor)

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