AAV2-regnase-1

AAV2-regnase-1 is a research-stage gene therapy construct consisting of an adeno-associated virus serotype 2 (AAV2) vector engineered to overexpress regnase-1, an endoribonuclease also known as MCPIP1 (encoded by the ZC3H12A gene). Regnase-1 acts as a key regulator of the inflammatory response by cleaving the mRNA of various pro-inflammatory cytokines, including IL-6, IL-8, and IL-12, leading to their degradation. In preclinical murine models, systemic delivery of AAV2-regnase-1—often utilizing modified capsids like AAV2-ESGHGYF for improved endothelial targeting—has demonstrated efficacy in reducing pulmonary arterial pressure and right ventricular hypertrophy in pulmonary hypertension. It has also been investigated for its potential to prevent the progression of thoracic aortic aneurysms in models of Marfan syndrome by reducing elastin degradation and aortic wall inflammation. The construct is primarily developed and studied by academic institutions, including the University of Kiel and the University of Heidelberg.

Other names

AAV2-MCPIP1AAV-2-MCPIP1AAV 2-MCPIP1AAV2-ZC3H12AAAV-2-ZC3H12AAAV 2-ZC3H12A