Viral gene therapy: Leveraging structural biology for safer cures

After decades of effort, gene therapy is happening: In November 2012, the European Commission approved a viral gene therapy for a rare metabolic syndrome causing pancreatitis, while clinical trials are well under way for a treatment for a rare form of blindness. Both therapies use adeno-associated viruses (AAVs) as a vector for delivering genes to the cell nucleus. But the pursuit of viral vectors for gene therapy and the battle against pathogenic viruses are both hampered by the subtle shape-shifting of viruses and the immune system’s equally dynamic response to them. Mavis Agbandje-McKenna will describe what structural virologists are doing to understand how AAVs recognize cell-surface receptors, traffic genes into the cell, fend off antibodies and then release and replicate in the nucleus.

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