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209 results

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Page 1
Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A.
Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Mahlangu J, et al. Among authors: pierce gf. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13. Blood. 2014. PMID: 24227821 Free PMC article. Clinical Trial.
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: pierce gf. Nat Med. 2006 Mar;12(3):342-7. doi: 10.1038/nm1358. Epub 2006 Feb 12. Nat Med. 2006. PMID: 16474400 Clinical Trial.
Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.
Jiang H, Couto LB, Patarroyo-White S, Liu T, Nagy D, Vargas JA, Zhou S, Scallan CD, Sommer J, Vijay S, Mingozzi F, High KA, Pierce GF. Jiang H, et al. Among authors: pierce gf. Blood. 2006 Nov 15;108(10):3321-8. doi: 10.1182/blood-2006-04-017913. Epub 2006 Jul 25. Blood. 2006. PMID: 16868252 Free PMC article.
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: pierce gf. 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.
Recombinant factor IX-Fc fusion protein (rFIXFc) demonstrates safety and prolonged activity in a phase 1/2a study in hemophilia B patients.
Shapiro AD, Ragni MV, Valentino LA, Key NS, Josephson NC, Powell JS, Cheng G, Thompson AR, Goyal J, Tubridy KL, Peters RT, Dumont JA, Euwart D, Li L, Hallén B, Gozzi P, Bitonti AJ, Jiang H, Luk A, Pierce GF. Shapiro AD, et al. Among authors: pierce gf. Blood. 2012 Jan 19;119(3):666-72. doi: 10.1182/blood-2011-07-367003. Epub 2011 Nov 22. Blood. 2012. PMID: 22110246 Free PMC article. Clinical Trial.
209 results