How Can We Develop Vectors That Enable Human Gene Therapy Using Models We Cannot Trust?
Time: 10:00 am
day: Day One AM
Details:
• Capsid engineering is a proven approach for generating AAV vectors with enhanced capabilities
• In vivo selections often lead to species-specific enhancements resulting in questions about translation
• Cross-model enhancements and mechanism of action studies provide increased confidence about the translation potential of engineered vectors