Syn-FLEX-CaMPARI AAV (Serotype 9)

In vivo gene therapy offers the possibility of a one-time cure for genetic diseases with the promise of lifelong beneficial effects. Gene therapy mediated by recombinant adeno-associated virus (rAAV) vectors has shown great promise in multiple clinical trials for neurological diseases, with sustained transgene expression and functional responses, as well as safety. While rAAV serotype 2 (rAAV2) has been the most widely used in clinical trials, many other serotypes have shown enhanced ability to transduce neurons in experimental studies. The adeno-associated virus serotype 9 (AAV9) vector has generated considerable interest in its therapeutic development for the treatment of neurological diseases, in part because of its perceived ability to cross the blood-brain barrier to target cells in the central nervous system (CNS). These cells include endothelial cells, neurons and astrocytes in the brain, and motor neurons and astrocytes in the spinal cord. The ability of AAV9 to target these CNS cells makes AAV9-mediated gene correction a viable therapy for ALD/AMN, as long bundles of CNS axons and their supporting glial cells are the site of pathology.