scAAV DJ-Cre

Adeno-associated virus (AAV) is a small (20 nm), replication-defective, non-pathogenic parvovirus. The single-stranded DNA (ssDNA) genome is up to 4.8 kb in size and is encapsidated in a non-enveloped capsid. Hundreds of serotypes and variants have been isolated and characterized, and they can infect both dividing and non-dividing cells in different tissues of humans and other animals. The Kay, Samulski, and Schaffer laboratories have constructed chimeric AAVs, such as AAV-DJ, through capsid evolution of AAV2, 8, and 9 for delivery of therapeutic genes to a variety of cell types. AAV has great potential as a gene delivery vector due to its advantages, such as minimal immunogenicity, long-term transgene expression, no clinically reported adverse events, high stability, and a wide range of cell types that can be infected. The first AAV-based gene therapy was approved by the European Medicines Agency (EMA) in 2012 for the treatment of lipoprotein lipase deficiency. The U.S. Food and Drug Administration (FDA) approved Luxturna and Zolgensma in 2017 and 2019, respectively, for the treatment of rare inherited forms of blindness and spinal muscular atrophy. There are currently more than 300 ongoing clinical studies for the treatment of various diseases or conditions, such as Alzheimer's disease, Parkinson's disease, cystic fibrosis, hemophilia B, HIV infection, Leber's congenital amaurosis, arthritis, and cancer. In addition to clinical applications, AAV is also an important research tool that can deliver reporter genes, CRISPR endonucleases, or other genes for basic research.