Strategies for development of novel adeno-associated viral vectors and production technology: towards national clinical-grade manufacturing capacity

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Gene therapy offers the possibility of new treatments for patients with genetic and acquired diseases. Modified non-pathogenic viruses can safely and efficiently deliver therapeutic DNA to affected cells inside a patient’s body, but manufacturing of these vectors is inefficient and expensive, hampering clinical development. This project focuses on further developing this technology to dramatically enhance manufacturing efficiency and build the foundations for an Australian production capacity.