Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII
Hu C, Cela RG, Suzuki M, Lee B, Lipshutz GS. Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc Natl Acad Sci U S A. 2011 Feb 1;108(5):2082-7. doi: 10.1073/pnas.1015571108. Epub 2011 Jan 18. PMID: 21245323; PMCID: PMC3033287.