Adeno-associated virus therapies: Pioneering solutions for human genetic diseases

Adeno-associated virus (AAV) has emerged as a fundamental component in the gene therapy landscape, widely acknowledged for its effectiveness in therapeutic gene delivery. The success of AAV-based therapies, such as Luxturna and Zolgensma, underscores their potential as a leading vector in gene therapy. This article provides an in-depth review of the development and mechanisms of AAV vector-based therapies, offering a comprehensive analysis of the latest clinical trial outcomes in central nervous system (CNS) diseases, ocular conditions, and hemophilia, where AAV therapies have shown promising results. Additionally, we discusse the selection of administration methods and serotypes tailored to specific diseases. Our objective is to showcase the innovative applications and future potential of AAV-based gene therapy, laying the groundwork for continued clinical advancements. • AAV is a cornerstone of gene therapy, proven by successful treatments like Luxturna and Zolgensma. • The review analyzes clinical trial results for AAV therapies, focusing on CNS diseases, ocular conditions, and hemophilia. • Choosing specific AAV serotypes and administration methods is key for targeting diseases, highlighting AAV gene therapy's future.