AAV PCSK9

AAV PCSK9 refers to a gene therapy approach that utilizes adeno-associated virus (AAV) vectors to modulate the expression of the Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9) gene. PCSK9 is a critical regulator of cholesterol homeostasis; it binds to the low-density lipoprotein receptor (LDLR) on the surface of hepatocytes, promoting its internalization and lysosomal degradation, which leads to elevated levels of circulating LDL cholesterol. Therapeutic AAV PCSK9 programs typically aim to achieve long-term reduction of LDL cholesterol by delivering genetic payloads—such as short hairpin RNA (shRNA), microRNA (miRNA), or gene-editing components (e.g., CRISPR/Cas9, ARCUS meganucleases)—that silence or permanently disrupt the PCSK9 gene in the liver. While several biopharmaceutical companies and academic institutions have explored AAV-mediated delivery for PCSK9 inhibition, many recent clinical efforts have transitioned toward lipid nanoparticle (LNP) delivery of mRNA-based editors to avoid the potential for long-term viral vector persistence and associated immunogenicity. Conversely, in preclinical research, AAV vectors are frequently used to overexpress gain-of-function variants of PCSK9 to induce hypercholesterolemia and atherosclerosis in animal models.

Other names

AAV-PCSK9AAV-PCSK-9AAV-PCSK 9AAV-mediated PCSK9 gene therapyAAV-shRNA-PCSK9AAV-shRNA-PCSK-9AAV-shRNA-PCSK 9AAV-CRISPR-PCSK9AAV-CRISPR-PCSK-9AAV-CRISPR-PCSK 9