Discovering the Next Generation AAV Vector Through Capsid Engineering and Expression Cassette Optimization

Learning Objectives:

1. Define the background introduction to AAV gene therapy

2. Recall the novel methods to build AAV capsid libraries for in vivo screening through rational design and directed evolution

3. Identify the tissue specific promoter and enhancer engineering to optimize transgene expression

4. Recall the design of a novel AAV-microdystrophin vector for Duchenne Muscular Dystrophy gene therapy