AAV6-MHCK7-GFP

Adeno-associated virus was first discovered in the 1960s, is replication-deficient, and belongs to the Parvoviridae family. AAV-based gene delivery systems are more attractive than other vectors. More benefits have been found using AAV vectors, such as greater safety due to lack of pathogenicity, more diverse host and cell type tropism, longer gene expression, ability to transfect both dividing and non-dividing cells, and no strong immune response. In addition, the discovery of more novel AAV serotypes will further expand the application scope of AAV-based gene delivery systems.

Various preclinical experiments involving AAV-based gene therapy have been conducted. In 1984, a study reported the first construction of AAV as a vector for gene transfer into eukaryotic cells, and the urgency of curing cystic fibrosis (CF) has driven the development of AAV-based in vivo gene therapy technology. Using AAV vectors, scientists successfully introduced a therapeutic gene, cystic fibrosis transmembrane regulator (CFTR), into the airways of rabbits and monkeys. The expression of CFTR was detected in the airways for up to 6 years. Subsequently, the first trial of AAV-mediated gene therapy for CF was reported in 1995. Since then, extensive research has been done on tissue tropism, targeting cell receptors associated with infection, gene/vector delivery routes, pathophysiologic effects, and AAV vector immune profiling. In recent years, gene therapy using different subtypes of AAV vectors has been reported for the treatment of a variety of diseases.