The Mechanism of Adeno-associated Virus (AAV) Gene Therapy

Adeno-associated Virus (AAV) vector has become an important tool for vivo gene therapy due to its high specificity, low immunogenicity and long-term expressibility. There are multiple novel pharmaceuticals utilizing recombinant AAV vectors to treat genetic defect disease or disease related to gene expression disorder that are undergoing clinical trials. In this review, the basic biological features of AAV viruses are introduced, including their structure, classification, and interactions with hosts. This paper aims to elucidate the commonalities and differences among several AAV viruses used in clinical treatment by analyzing existing literature. The relevant genes for transduction of various types of AAV viruses are specifically analyzed in this article. By elaborating the process and mechanism of AAV virus transduction-related gene expression, this paper also attempts to summarize the current achievements of AAV therapy and explain the limitations still existing. It is hoped to contribute to the development and progress of AAV therapy in the future.