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Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response.
Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, Rasko JJ, Ozelo MC, Hoots K, Blatt P, Konkle B, Dake M, Kaye R, Razavi M, Zajko A, Zehnder J, Rustagi PK, Nakai H, Chew A, Leonard D, Wright JF, Lessard RR, Sommer JM, Tigges M, Sabatino D, Luk A, Jiang H, Mingozzi F, Couto L, Ertl HC, High KA, Kay MA. Manno CS, et al. Among authors: jiang h. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
CD8(+) T-cell responses to adeno-associated virus capsid in humans.
Mingozzi F, Maus MV, Hui DJ, Sabatino DE, Murphy SL, Rasko JE, Ragni MV, Manno CS, Sommer J, Jiang H, Pierce GF, Ertl HC, High KA. Mingozzi F, et al. Among authors: jiang h. Nat Med. 2007 Apr;13(4):419-22. doi: 10.1038/nm1549. Epub 2007 Mar 18. Nat Med. 2007. PMID: 17369837
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B.
Arruda VR, Stedman HH, Haurigot V, Buchlis G, Baila S, Favaro P, Chen Y, Franck HG, Zhou S, Wright JF, Couto LB, Jiang H, Pierce GF, Bellinger DA, Mingozzi F, Nichols TC, High KA. Arruda VR, et al. Among authors: jiang h. Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24. Blood. 2010. PMID: 20335222 Free PMC article.
Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B.
Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Powell JS, et al. Among authors: jiang h. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4. N Engl J Med. 2013. PMID: 24304002 Free article. Clinical Trial.
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