Adenine base editing as a promising therapy for cardiovascular diseases

Abstract

Cardiovascular diseases (CVDs) are the leading causes of human death worldwide. Genetic variants serve as the major risk factor for CVDs, with limited therapeutic interventions in clinical practice. The recent surge of genome editing technologies offers the hope to correct genetic variants and to cure genetic diseases. Among the diverse genome editing tools, adenine base editors (ABEs) exhibit high efficiency, high specificity, and low off-target effects, successfully entering a clinical trial and demonstrating the tremendous potential to transform modern cardiovascular therapy. In this review, we summarize the basic knowledge about ABE, showcase three hallmark studies using ABE to ameliorate or treat CVDs in experimental animals, and lastly discuss about the key technical concerns that should be addressed to achieve the full potential of ABEs in the future.