Senior Director of Gene Transfer Technologies, REGENXBIO Inc.
BIOGRAPHY
JUL 22, 2021 4:15 PM EDT
Discovering the Next Generation AAV Vector Through Capsid Engineering and Expression Cassette Optimization
Presented at:
GenScript Gene & Cell Engineering Virtual Summit
Speaker
Abstract
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
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