CAG-FLEX-jGCaMP7c AAV (Serotype 9)

The field of gene therapy has made significant progress over the past few decades, and there are now FDA-approved treatments for a variety of genetic diseases that were once considered untreatable. Adeno-associated virus (AAV) is one of the most effective vectors for delivering healthy gene cDNA sequences into the cells of diseased individuals due to its ability to persist as free bodies in the nucleus for long periods of time, its relative lack of pathogenicity, and its ability to infect both dividing and non-dividing cells in vivo. Of all the AAV serotypes and variants tested to date, AAV9 has been popular for gene therapy applications due to its high tropism for tissues such as liver, heart, and skeletal muscle after intravascular (IV) delivery. In addition, its ability to cross the blood-brain barrier (BBB) ​​and transduce cells residing in the central nervous system has opened up new avenues for CNS gene therapy. For example, in a mouse model of ASPA deficiency (Canavan disease, a white matter neurodegenerative disorder), intravenous administration of rAAV9 expressing the human ASPA gene was effective in rescuing most disease phenotypes. Recently, clinical trials for the treatment of spinal muscular atrophy via intravenous administration of rAAV9 have shown promising early results. In addition to pursuing enhanced transduction in target tissues following intravenous administration of rAAV, targeting specific tissues is often a desirable feature to reduce potential off-target toxicities.