CAG-GCaMP3 AAV (Serotype 9)

The discovery that AAV9 can cross the blood-brain barrier (BBB) was the starting point for extensive investigation of its therapeutic effects in a variety of central nervous system (CNS) diseases, of which intravenous administration is the main route of administration. In general, AAV9 is used to repair defective genes, as in the case of lysosomal storage diseases and spinal muscular atrophy type 1; to silence dominantly inherited mutant alleles, as in Huntington's disease; or to introduce disease-modifying genes that may alleviate neuropathology, as in the study of Alzheimer's disease. Theoretically, given the natural properties of vascular AAV9-mediated gene therapy, the most suitable candidates for vascular AAV9-mediated gene therapy are diseases that: i) have a clear genetic cause, so the affected gene can be directly targeted; ii) present with early onset, which is conducive to vector administration in childhood, when neuronal tropism may be stronger; iii) affect both the CNS and peripheral organs, which is compatible with the natural AAV9 biodistribution.