scAAV DJ/8-CAG-GFP

The self-complementary adeno-associated virus (scAAV) serotype DJ/8 represents a significant advancement in viral vector technology for gene therapy applications. scAAV DJ/8 combines two key features: a self-complementary genome structure and an engineered DJ/8 capsid. The self-complementary design allows it to immediately form double-stranded DNA upon entering the cell, bypassing the rate-limiting step of second-strand synthesis required by traditional single-stranded AAV vectors. This results in faster and more efficient transgene expression. Compared to natural AAV serotypes, the DJ/8 serotype is particularly noteworthy for its broader tissue tropism and higher transduction efficiency in a variety of cell types. Engineered through capsid recombination, DJ/8 exhibits enhanced cell entry and nuclear transport capabilities while maintaining the low immunogenicity characteristic of AAV vectors. These properties make scAAV DJ/8 an extremely powerful gene delivery tool, capable of achieving higher expression levels at lower vector doses compared to traditional AAV vectors.

The scAAV DJ/8 vector system has been widely applied in research and therapeutic development. In basic research, it effectively facilitates gene transfer in various animal models, particularly suitable for studying diseases requiring rapid and high-level transgene expression, such as neurological disorders, metabolic diseases, and muscular dystrophy. In therapeutic applications, scAAV DJ/8 holds promise for treating liver diseases, retinal diseases, and central nervous system disorders due to its ability to cross endothelial barriers and transduce difficult-to-target tissues. Its enhanced transduction capabilities in skeletal and cardiac muscle also make it valuable in the treatment of muscle diseases.