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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12.
Nat Med. 2006.
PMID: 16474400
Clinical Trial.
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.
Manno CS, Chew AJ, Hutchison S, Larson PJ, Herzog RW, Arruda VR, Tai SJ, Ragni MV, Thompson A, Ozelo M, Couto LB, Leonard DG, Johnson FA, McClelland A, Scallan C, Skarsgard E, Flake AW, Kay MA, High KA, Glader B.
Manno CS, et al.
Blood. 2003 Apr 15;101(8):2963-72. doi: 10.1182/blood-2002-10-3296. Epub 2002 Dec 19.
Blood. 2003.
PMID: 12515715
Free article.
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