Non-viral Gene Therapy for Hemophilia A Through Oral Delivery of Chitosan Nanoparticles

摘要

Oral delivery of factor VIII DNA in chitosan nanoparticlesresults in tissue distribution of plasmid DNA to liver,spleen, stomach, and ileum. While plasma protein levelsare very low, functional FVIII is detected through enhancedsurvival in a phenotypic bleeding challenge.