Report Summary

Direct Comparative Analysis of rAAV1 and rAAV5 Pseudotyped Vectors Using Aerosol Delivery of Firefly and Renila Luciferase Reporters Co-delivered to the Lungs of Chimpanzees

Study Summary

Overall Study Design 
The selection of the most efficient rAAV capsid serotype for airway gene therapy of cystic fibrosis CF has been challenging 
due to cross-specific differences in tropism between humans and animal models Additionally species-specific differences 
in the immune response profile to various rAAV serotypes can also greatly impact the outcome of clinical trials Investigators 
postulated that the use of chimpanzees which are the closest surviving genetic relative to humans could be the most 
predictive model for selection of a rAAV serotype best suited for human trials in CF patients 
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