Syn-CaMPARI AAV (Serotype 9)

Recombinant adeno-associated virus (rAAV) vectors have been widely used in preclinical and clinical research for decades. Some AAV-based gene therapies have been approved for marketing in the United States and Europe, and several other promising AAV-mediated gene therapy drugs are entering Phase III clinical trials. rAAV vectors are also considered a promising platform for the treatment of central nervous system (CNS) diseases. However, because AAV vectors cannot effectively cross the blood-brain barrier (BBB), AAV-mediated gene therapy for CNS diseases must be achieved through intraparenchymal injection or high-dose systemic administration. Although intraparenchymal injection can successfully deliver AAV vectors to localized areas of the brain, most neurodegenerative diseases, such as amyotrophic lateral sclerosis, Huntington's disease, leukodystrophy, and lysosomal storage diseases, involve cell damage in multiple regions rather than a single site. Studies have shown that some AAV serotypes, such as AAV7, AAV8, AAV9, AAVrh.8, and AAVrh.10, have the ability to transduce neurons and other types of brain cells, and the best of these is AAV9. AAV9 has an excellent ability to transduce neurons, brain parenchymal cells, and some BBB endothelial cells. It is considered to be a very promising AAV vector for central nervous system diseases.