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2012 | 1 |
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AAV-based neonatal gene therapy for hemophilia A: long-term correction and avoidance of immune responses in mice.
Gene Ther. 2012 Dec;19(12):1166-76. doi: 10.1038/gt.2011.200. Epub 2012 Jan 12.
Gene Ther. 2012.
PMID: 22241178
Free PMC article.
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.
Hu C, et al.
Proc Natl Acad Sci U S A. 2011 Feb 1;108(5):2082-7. doi: 10.1073/pnas.1015571108. Epub 2011 Jan 18.
Proc Natl Acad Sci U S A. 2011.
PMID: 21245323
Free PMC article.
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