AAV PHP.S-CMV-GFP

Gene therapy is an emerging and promising therapeutic strategy that has been applied to a variety of diseases, including neurodegenerative diseases, monogenic diseases, and tumors. More than 2,000 clinical trials have safely used gene therapy for treatment. One of the most promising gene delivery tools is the adeno-associated virus (AAV) vector, which can transduce dividing and non-dividing cells, is non-pathogenic, and can achieve stable and efficient gene expression. In recent years, AAV-based gene therapy has shown good prospects in the treatment of various central nervous system diseases, and many AAV-mediated therapeutics have been used in clinical trials to treat diseases such as Alzheimer's disease, Parkinson's disease, and Huntington's disease.

Compared with naturally occurring capsids, the novel AAV-PHP capsids show significantly improved tropism for cells in the central nervous system (CNS), peripheral nervous system (PNS), and visceral organs of adult mice. AAV-PHP.B and the further evolved AAV-PHP.eB effectively transduce neurons and glial cells throughout the central nervous system; a second variant, AAV-PHP.S, shows enhanced tropism for neurons within the peripheral nervous system and organs such as the gut and heart. Importantly, these capsids target cell populations that are typically difficult to reach because of their location (e.g., sympathetic nerves, tuberosity, dorsal root and cardiac ganglia) or their widespread distribution (e.g., throughout the brain or enteric nervous system). Along with the capsid, the genetic cargo (or AAV genome) can be customized to control transgene expression.