AAV9-Syn-iCre

rAAV was first produced in the early 1980s. rAAV contains antibiotic resistance genes that can replace caps to successfully transduce mammalian cells, thus establishing the potential of AAV as a gene delivery vector. In December 2017, the FDA approved the first gene therapy for the treatment of a genetic disease, an AAV2-based product for the treatment of RPE65-mediated inherited retinal dystrophy. rAAV has several key properties that make it promising for gene therapy. Its genome and capsid structure are relatively simple, and the ITRs are the only cis-acting elements required to package the transgene into the capsid. In addition, AAV is non-pathogenic and has relatively low immunogenicity and genotoxicity. Serotypes exhibit a variety of tropisms and immune response characteristics suitable for different applications. AAV8 preferentially targets the liver, while cardiac and skeletal muscle gene transfer appears to be best mediated by AAV1, -6, and -9. AAV9 and AAVrh.10 have demonstrated the ability to cross the blood-brain barrier (BBB) ​​when injected intravenously. Notably, although AAV exists primarily in a free form, transgene expression can be detected up to 10 years after AAV injection.