AAV5-CAG-ZsGreen

Thanks to the rapid development of biomedical technology, new high-tech treatments for a variety of human diseases are now widely available. However, despite the many scientific discoveries in the field of gene and cell therapy, some diseases still cannot be effectively treated. Advances in genetic engineering methods have enabled the development of effective gene therapy methods for a variety of diseases based on adeno-associated viruses (AAV). AAV is effectively used to treat a variety of inherited and acquired human diseases, more specifically, monogenic inherited diseases, i.e. diseases caused by mutations in a single gene. Today, many AAV-based gene therapy drugs are being studied in preclinical and clinical trials, and new drugs are available on the market for the treatment of a wide range of human diseases, including those that were previously considered untreatable. Depending on the serotype, AAV can be specific for specific organs and tissues of the body. Different AAV serotypes differ in many aspects. Similar to AAV1 and AAV4, mucin columns can be used for AAV5 purification, and ion exchange chromatography techniques have been developed. It has been reported that the capsid protein of rAAV5 can undergo a variety of post-translational modifications (PTMs), including ubiquitination, phosphorylation, SUMOylation, and glycosylation. AAV5 has been shown to have a remarkable transduction efficiency for mouse retinal cells, primarily photoreceptors. In addition, the tropism of AAV5 has been studied in the mouse brain and has been shown to be able to transduce a variety of neural cell types, including Purkinje cells, stellate neurons, basket neurons, and Golgi neurons, as well as reach the hypothalamus and ventricular epithelium. AAV5 is also known for its high efficiency in transducing mouse airway epithelium, vascular endothelial cells, and smooth muscle via apical infection. It has also been reported to have a tropism for mouse hepatocytes.