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Gene therapy to treat human disease has evolved from a relatively small group of dedicated scientists working on the fundamentals of gene delivery to mammalian cells, to a large industry capable of ex-vivo and in vivo gene therapy in patients. While many promising delivery systems exist or are being developed, adeno-associated virus (AAV) vectors are currently the most tested and most efficient vehicle for in vivo human gene therapy. However, clinical data highlights the need for improved delivery systems.
This webinar will focus on building novel AAV vectors to overcome some of these challenges, which include inefficient delivery to target organs and immune evasion. Specifically, the use of improved AAV library systems, AAV capsid repurposing, and the use of extracellular vesicles to enhance desired vector properties will be covered.
Associate Professor of Neurology, Harvard Medical School and Investigator, Massachusetts General Hospital
Casey Maguire, PhD is Associate Professor of Neurology at Harvard Medical School and an Investigator at the Massachusetts General Hospital. His research focuses on improving virus vectors to allow effective human gene therapy in the face of delivery and immune-related barriers.
