Syn-FLEX-NES-jRCaMP1b AAV (Serotype 9)

Over the past decade, adeno-associated virus (AAV) vectors have emerged as the most promising gene delivery system for human gene therapy. By replacing the rep and cap genes with a therapeutic gene of interest (GOI), the resulting recombinant AAV-derived vectors (rAAV) can be safely and effectively used to deliver target genes to a variety of human tissues with long-term gene expression. The safety of AAV is attributed to its requirement for a helper virus for replication, and many AAV serotypes have a range of tissue tropisms.

Recombinant AAV vectors can transduce a variety of tissues in vivo and provide long-term gene expression without eliciting significant cell-mediated immune responses. A number of rAAV-based gene therapies have been approved by the U.S. Food and Drug Administration (FDA) for clinical treatment of retinal dystrophy, spinal muscular atrophy, hemophilia B, and Duchenne muscular dystrophy, highlighting the growing importance of rAAV in this field. To date, 4,100 AAV serotypes have been reported. Comparison of recently discovered serotypes showed that AAV-9 transduced the heart, lung, and tibialis anterior (TA) muscle better than all other serotypes after intravenous (IV) injection and was independent of age, whereas transduction into the liver and kidney was age-dependent.