Research Status and Applications of Adeno-Associated Virus.

Abstract

Adeno-associated virus (AAV) has emerged as a powerful and effective tool for the delivery of exogenous genes into various cells or tissues. To improve the gene delivery efficiency, as well as the safety and specificity of AAV's cell-targeting capabilities, extensive investigations have been conducted into its molecular biological characteristics, including capsid structure, cellular tropism, and the mechanisms underlying its entry, replication, DNA packaging, and capsid assembly. Significant differences exist between human and non-human primate AAVs regarding tissue targeting and transduction efficiency. These differences are primarily attributed to the amino acid sequences of AAV capsid proteins, the structural characteristics of these proteins, and the interactions of AAV with surface factors on host cells, such as cell surface receptors, signaling molecules, and associated proteins. This review primarily focuses on several key aspects of AAV, including its genome, coat proteins and their structures, genome replication, virus assembly, and the role of helper viruses. Additionally, it examines the utilization of recombinant adeno-associated viruses (rAAV), detailing their production methods, mechanisms of cell entry and trafficking, and various serotypes. The review further interprets the role of rAAV by analyzing its current applications in research and therapy.