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

Gene therapy offers a promising treatment for genetic diseases. With the rapid development of gene delivery vectors, the delivery method plays a crucial role in gene therapy. Among various gene therapy delivery vectors, adeno-associated virus (AAV) meets the criteria of high efficiency and non-pathogenicity as a human viral vector. To date, there are several AAV-based gene therapies approved by the U.S. Food and Drug Administration (FDA), as well as a large number of ongoing clinical trials.

Recently discovered adeno-associated viral vector (AAV) serotypes that can cross the blood-brain barrier (BBB) ​​have produced unprecedented therapeutic effects in animal models of neurological diseases. AAV9 is the first serotype that can effectively cross the BBB. Subsequently, multiple research groups have demonstrated that intravenous injection of AAV9 into mice, rats, cats, and non-human primates results in robust transduction in the central nervous system (CNS). Intravenous injection of AAV9 into newborn mice primarily results in neuronal transduction throughout the brain and spinal cord, but preferential targeting of astrocytes occurs when injected into mice older than 2 weeks. Similar patterns of CNS transduction have been reported for other AAV serotypes after systemic administration. The affinity of AAV for neural tissue suggests that the peripheral nervous system may be transduced after systemic injection.