CMV-GFP AAV (Serotype BR1)

Adeno-associated viruses (AAVs) are non-enveloped viruses with an icosahedral capsid protein coat that encloses a single-stranded DNA genome (approximately 4.7 kilobases). The capsid protein coat is composed of 60 monomeric viral protein (VP) subunits, VP1, VP2, and VP3, in a 1:1:10 ratio, respectively. The capsid protein coat provides shielding for the AAV genome and determines the tissue tropism of A​​AV administered in vivo. After homing to an organ or tissue, the capsid proteins promote internalization of AAV into host tissue cells and regulate the subsequent intracellular trafficking of AAV to the nucleus. Intracellular trafficking of AAV involves binding of AAV to cell surface receptors, followed by internalization by endocytosis and subsequent entry into the nucleus. AAV uncoats in the nucleus and releases its genome into the host cell nucleus. The probability of AAV genome integration into the host cell genome (chromosomal integration) is only ~ 0.1%.

AAV-BR1 is a capsid-modified AAV vector known for its strong tropism for brain endothelial cells (BECs) and, when injected intravenously, has been shown to produce durable transgene expression in the brain with minimal transduction of peripheral organs except the lung. The therapeutic potential of AAV-BR1 has recently been demonstrated in different mouse models of neurodegenerative diseases, such as Incontinentia pigmenti, Sandhoff disease, and Allan-Herndon-Dudley syndrome, which can be successfully treated by altering gene expression in BECs.