Gene therapy has the potential to treat severe diseases by delivering therapeutic genes to the patient’s cells. Recombinant adeno-associated virus (rAAV) is the most popular vector for in vivo gene delivery, due to its relatively low immunogenicity, broad tissue tropism, and long-term gene expression capabilities. Recently, FDA has approved an increasing number of rAAV-based gene therapies for the treatment of various conditions, including genetic diseases that result in blindness, muscular dystrophy, and blood disorders. The greatest challenge in cell culture-based production of rAAV is the large number of empty vectors, lacking the therapeutic transgene. These empty vectors can comprise 50-90% of the total AAV produced. Yet few methods exist to reliably increase the percentage of filled capsids produced by current culture-based production systems. This deficit in packaging capabilities Gene therapy has the potential to treat severe diseases by delivering therapeutic genes to the patient’s cells. Recombinant adeno-associated virus (rAAV) is the most popular vector for in vivo gene delivery, due to its relatively low immunogenicity, broad tissue tropism, and long-term gene expression capabilities. Recently, FDA has approved an increasing number of rAAV-based gene therapies for the treatment of various conditions, including genetic diseases that result in blindness, muscular dystrophy, and blood disorders. The greatest challenge in cell culture-based production of rAAV is the large number of empty vectors, laccan lead to adverse immunological responses and increase manufacturing costs. The project aims to develop novel approaches to improving the in vitro packaging capacity by identifying the essential viral elements.

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