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Novel hemophilia B mouse models exhibiting a range of mutations in the Factor IX gene.

Sabatino DE, Armstrong E, Edmonson S, Liu YL, Pleimes M, Schuettrumpf J, Fitzgerald J, Herzog RW, Arruda VR, High KA.

Blood. 2004 Nov 1;104(9):2767-74. Epub 2004 Jun 24.


Creation of a mouse expressing defective human factor IX.

Jin DY, Zhang TP, Gui T, Stafford DW, Monahan PE.

Blood. 2004 Sep 15;104(6):1733-9. Epub 2004 Jun 3.


Targeted inactivation of the coagulation factor IX gene causes hemophilia B in mice.

Kundu RK, Sangiorgi F, Wu LY, Kurachi K, Anderson WF, Maxson R, Gordon EM.

Blood. 1998 Jul 1;92(1):168-74.


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.

Blood. 2003 Apr 15;101(8):2963-72. Epub 2002 Dec 19.


Major role of local immune responses in antibody formation to factor IX in AAV gene transfer.

Wang L, Cao O, Swalm B, Dobrzynski E, Mingozzi F, Herzog RW.

Gene Ther. 2005 Oct;12(19):1453-64.


Factor IX variants improve gene therapy efficacy for hemophilia B.

Schuettrumpf J, Herzog RW, Schlachterman A, Kaufhold A, Stafford DW, Arruda VR.

Blood. 2005 Mar 15;105(6):2316-23. Epub 2004 Nov 18.


Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

Fields PA, Arruda VR, Armstrong E, Chu K, Mingozzi F, Hagstrom JN, Herzog RW, High KA.

Mol Ther. 2001 Sep;4(3):201-10.


Theodore E. Woodward Award. AAV-mediated gene transfer for hemophilia.

High KA.

Trans Am Clin Climatol Assoc. 2003;114:337-51; discussion 351-2. Review.


Molecular genotyping of the Italian cohort of patients with hemophilia B.

Belvini D, Salviato R, Radossi P, Pierobon F, Mori P, Castaldo G, Tagariello G; AICE HB Study Group.

Haematologica. 2005 May;90(5):635-42.


Transgene expression levels and kinetics determine risk of humoral immune response modeled in factor IX knockout and missense mutant mice.

Zhang TP, Jin DY, Wardrop RM 3rd, Gui T, Maile R, Frelinger JA, Stafford DW, Monahan PE.

Gene Ther. 2007 Mar;14(5):429-40. Epub 2006 Oct 26.


Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX.

Fields PA, Kowalczyk DW, Arruda VR, Armstrong E, McCleland ML, Hagstrom JN, Pasi KJ, Ertl HC, Herzog RW, High KA.

Mol Ther. 2000 Mar;1(3):225-35.


Molecular analysis of hemophilia B in Poland: 12 novel mutations of the factor IX gene.

Wulff K, Bykowska K, Lopaciuk S, Herrmann FH.

Acta Biochim Pol. 1999;46(3):721-6.


Hepatic control elements promote long-term expression of human coagulation factor IX gene in hydrodynamically transfected mice.

Kim HS, Kim JC, Lee YK, Kim JS, Park YS.

J Gene Med. 2011 Jul;13(7-8):365-72. doi: 10.1002/jgm.1583.


[Establishment of a hemophilia B transgenic mouse model on the basis of coagulation factor IX gene knock-out mouse].

Che WL, He Y, Yao ZZ, Li J, Fu JL.

Yi Chuan Xue Bao. 2002 Jul;29(7):594-8. Chinese.


Technology evaluation: AAV factor IX gene therapy, Avigen Inc.

Fabb SA, Dickson JG.

Curr Opin Mol Ther. 2000 Oct;2(5):601-6. Review.


In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs.

Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR, et al.

Proc Natl Acad Sci U S A. 1994 Mar 15;91(6):2353-7.


Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

Kay MA, Manno CS, Ragni MV, Larson PJ, Couto LB, McClelland A, Glader B, Chew AJ, Tai SJ, Herzog RW, Arruda V, Johnson F, Scallan C, Skarsgard E, Flake AW, High KA.

Nat Genet. 2000 Mar;24(3):257-61.


Therapeutic plasma concentrations of human factor IX in mice after gene delivery into the amniotic cavity: a model for the prenatal treatment of haemophilia B.

Schneider H, Adebakin S, Themis M, Cook T, Douar AM, Pavirani A, Coutelle C.

J Gene Med. 1999 Nov-Dec;1(6):424-32.


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