91桃色

Abstract

Adeno-associated virus (AAV) vectors have emerged as one of the most promising tools in gene therapy, particularly for inherited liver disorders. These disorders, including hemophilia, are caused by genetic mutations that prevent the liver from producing necessary clotting factors, leading to serious health complications. AAV vectors offer an innovative approach to gene delivery, allowing for the targeted correction of genetic defects in the liver. This article explores the role of AAV vectors in treating inherited liver disorders, with a particular focus on hemophilia. We discuss the mechanism of AAV-based gene therapy, its advantages, the clinical challenges associated with AAV vector therapy, and recent advances that are making these treatments increasingly viable. Additionally, we highlight the potential of AAV vectors to revolutionize the management of genetic liver disorders, offering hope for more effective, long-lasting therapies.