If you've already registered, please click here to log in to the webcast

Recombinant AAV vectors have become more popular for in vivo based gene therapy approaches. There are now two FDA approved AAV drugs and more likely to be approved in the next few years. In this webcast, Dr. Mark Kay, a research leader in AAV gene therapy approaches, will describe current limitations and how his Stanford University lab is working to address them.

Some of the limitations include the inability to predict which AAV serotype will work best in humans based on animal studies. The speaker will describe use of humanized murine models in an attempt to make improved predictions as well as discover more effective AAV serotypes.

In addition, AAV-mediated gene transfer will not persist in regenerating or growing tissues, making it difficult to treat diseases with early onset phenotypes. Dr. Kay’s group has used AAV vectors to induce non-nuclease mediated homologous recombination to add a therapeutic coding sequence onto an endogenous gene without disrupting its function, allowing for stable and life-long expression. This approach has been used to treat several mouse models of human disease. The researchers hope that improving AAV vectors will also expand their use to include a myriad of disorders that require treatment at or near birth.

You will learn

• Why AAV vectors are such commonly used gene transfer vehicle for in vivo gene therapy approaches
• Current limitations using AAV vectors
• Challenges moving from preclinical animal studies to humans