Report Summary

Progress with Recombinant Adeno-Associated Virus Vectors for Gene Therapy of Alpha-1 Antitrypsin Deficiency

Study Summary

Overall Study Design 
The pathway to a clinical gene therapy product often involves many changes of course and strategy before obtaining 
successful results Here we outline the methodologies both clinical and preclinical that went into developing a gene 
therapy approach to the treatment of alpha-1 antitrypsin deficiency lung disease using muscle-targeted recombinant adeno-
associated virus From initial gene construct development in mouse models through multiple rounds of safety and 
biodistribution studies in rodents rabbits and nonhuman primates to ultimate human trials this review seeks to provide 
insight into what clinical translation entails and could thereby inform the process for future investigators 
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