CAG-ChIEF-tdTOMATO AAV (Serotype 9)

Adeno-associated virus (AAV) vectors have great potential for gene therapy applications due to their ability to transduce many different types of tissues and provide stable, long-term gene expression. Multiple serotypes of AAV have been identified, and more than 120 additional variants have been isolated from human and non-human primate tissues. Each of these variants has unique properties, providing a large number of potential vectors for gene therapy. Different AAV serotypes exhibit different tissue tropisms and transduction efficiencies. For example, AAV1 exhibits efficient muscle transduction, AAV6 shows promise for targeting the lung conducting airway epithelium, and AAV8 has impressive liver transduction.

The AAV serotype with the most interesting properties as a vector is the AAV based on the human-derived virus AAV9. Following intravenous injection, AAV9 exhibits broad systemic transduction, including efficient targeting of the heart, liver, and skeletal muscle. Recently, AAV9 has been shown to transduce multiple different cell types in vitro and mouse airway cells in vivo using galactose as a receptor. In addition, AAV9 is able to cross the blood-brain barrier and transduce cells such as neurons in the brain and spinal cord. Therefore, rAAV9 is a potential therapeutic vector for the treatment of cardiac, neurodegenerative and neuromuscular diseases.