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Gene therapy addresses the underlying genetic anomaly by correcting the mutation using gene-editing tools deployed via delivery vehicles. Recombinant viral vectors such as lentiviruses or adeno associated viruses (AAVs), have been successfully employed as vehicles for gene therapy in clinical trials over the last decades. However, the complexity of the viral vector structure, mechanisms of viral-host interactions, delivery barriers, and optimal dosages compounded by the reported adverse risks, has paved the way to enhance existing vectors and pioneer novel approaches to deliver the next-generation of viral vectors.
In this GEN webinar, our distinguished guests, Dr. Arun Srivastava and Dr. David Schaffer will tell us more about advances in viral vector development and research enabling effective gene therapies that are reliable, safer, and potent. Specifically, our speakers will highlight information on a novel serotype vector exhibiting high transduction efficiency of hematopoietic stem cells to treat blood disorders. Furthermore, we will learn more about hybrid vectors with highly efficient nuclease-free genome editing solution, in addition to how the directed evolution of AAV vectors is improving the delivery efficiency to multiple organs with the ability to evade immune response.
A live Q&A followed the presentation, offering a chance to pose questions to our expert panelists.
Professor of Genetics
Chief, Division of Cellular and Molecular Therapy
University of Florida College of Medicine
Professor; Director of QB3
University of California, Berkeley
Produced with support from:
