GFP Adeno-Associated Virus ( AAV-LSPVR )

One of the earliest studies demonstrating that recombinant AAV vectors could be used for gene transfer in the mammalian heart was conducted using AAV serotype 2. Long-term expression of the lacZ reporter gene was observed 2 months after direct intramyocardial injection in rat hearts and up to 6 months after intracoronary infusion in adult pigs. Later studies reported significantly improved transduction efficiency following intracoronary infusion of recombinant AAV2 vectors in adult mice. After more than a decade of evaluation of recombinant AAV vectors as a potential tool for cardiac gene transfer, the first phase I/II clinical trial using AAV1 vectors for the treatment of heart failure was designed. This study involved intracoronary infusion of AAV1 vectors packaged with the SERCA2a gene, which encodes a major cardiac calcium cycling protein. GFP AAV-LSPVR particles are derived from AAV serotype 2 (AAV2), a commonly used serotype known for its transduction efficiency in a variety of cell types. For AAV-LSPVR, the capsid was specifically modified, including the insertion of the peptide sequence LSPVRPG at amino acid position 588 (I588). This modification was designed to optimize the viral vector for specific interactions and transduction capabilities. One of the most notable features of GFP AAV-LSPVR is its targeted infectivity to cardiomyocytes (precursor cells that differentiate into cardiomyocytes). Cardiomyocytes play a key role in heart development and function, and their precise targeting is critical for research involving cardiac gene therapy and regenerative medicine.