recombinant adeno-associated virus 2

Recombinant adeno-associated virus 2 (rAAV2) is a widely utilized viral vector platform in gene therapy, derived from a non-pathogenic parvovirus. It is engineered by replacing the viral protein-coding genes (rep and cap) with a therapeutic transgene expression cassette, while retaining the inverted terminal repeats (ITRs) required for packaging. rAAV2 is characterized by its ability to mediate long-term transgene expression in both dividing and non-dividing cells and exhibits a natural tropism for tissues such as the retina, central nervous system, and liver. Its primary mechanism of cellular entry involves binding to heparan sulfate proteoglycan (HSPG) receptors on the cell surface. rAAV2 has been successfully employed in clinical applications, most notably as the vector for the first FDA-approved AAV-based gene therapy, voretigene neparvovec. Recent research focuses on optimizing its manufacturing productivity, including the use of additives like dimethyl sulfoxide (DMSO) to improve full capsid yields and the implementation of process analytical technology (PAT) for real-time monitoring of cell-state dynamics during transient transfection in HEK293 cells.

Other names

AAV2AAV-2AAV 2adeno-associated virus 2adeno-associated virus serotype 2