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Pharmacological modulation of humoral immunity in a nonhuman primate model of AAV gene transfer for hemophilia B.

Mingozzi F, Chen Y, Murphy SL, Edmonson SC, Tai A, Price SD, Metzger ME, Zhou S, Wright JF, Donahue RE, Dunbar CE, High KA.

Mol Ther. 2012 Jul;20(7):1410-6. doi: 10.1038/mt.2012.84. Epub 2012 May 8.


Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

Mingozzi F, Hasbrouck NC, Basner-Tschakarjan E, Edmonson SA, Hui DJ, Sabatino DE, Zhou S, Wright JF, Jiang H, Pierce GF, Arruda VR, High KA.

Blood. 2007 Oct 1;110(7):2334-41. Epub 2007 Jul 3.


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.


Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy.

Herzog RW, Fields PA, Arruda VR, Brubaker JO, Armstrong E, McClintock D, Bellinger DA, Couto LB, Nichols TC, High KA.

Hum Gene Ther. 2002 Jul 20;13(11):1281-91.


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.


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.

Blood. 2006 Nov 15;108(10):3321-8. Epub 2006 Jul 25.


Improved induction of immune tolerance to factor IX by hepatic AAV-8 gene transfer.

Cooper M, Nayak S, Hoffman BE, Terhorst C, Cao O, Herzog RW.

Hum Gene Ther. 2009 Jul;20(7):767-76. doi: 10.1089/hum.2008.161.


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.


Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

Arruda VR, Schuettrumpf J, Herzog RW, Nichols TC, Robinson N, Lotfi Y, Mingozzi F, Xiao W, Couto LB, High KA.

Blood. 2004 Jan 1;103(1):85-92. Epub 2003 Sep 11.


Role of the vector genome and underlying factor IX mutation in immune responses to AAV gene therapy for hemophilia B.

Rogers GL, Martino AT, Zolotukhin I, Ertl HC, Herzog RW.

J Transl Med. 2014 Jan 25;12:25. doi: 10.1186/1479-5876-12-25.


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.


Muscle as a target for supplementary factor IX gene transfer.

Hoffman BE, Dobrzynski E, Wang L, Hirao L, Mingozzi F, Cao O, Herzog RW.

Hum Gene Ther. 2007 Jul;18(7):603-13.


Transient and intensive pharmacological immunosuppression fails to improve AAV-based liver gene transfer in non-human primates.

Unzu C, Hervás-Stubbs S, Sampedro A, Mauleón I, Mancheño U, Alfaro C, de Salamanca RE, Benito A, Beattie SG, Petry H, Prieto J, Melero I, Fontanellas A.

J Transl Med. 2012 Jun 15;10:122. doi: 10.1186/1479-5876-10-122.


Role of antigen-specific regulatory CD4+CD25+ T cells in tolerance induction after neonatal IP administration of AAV-hF.IX.

Shi Y, Falahati R, Zhang J, Flebbe-Rehwaldt L, Gaensler KM.

Gene Ther. 2013 Oct;20(10):987-96. doi: 10.1038/gt.2013.22. Epub 2013 Jun 13.


Persistent expression of hF.IX After tolerance induction by in utero or neonatal administration of AAV-1-F.IX in hemophilia B mice.

Sabatino DE, Mackenzie TC, Peranteau W, Edmonson S, Campagnoli C, Liu YL, Flake AW, High KA.

Mol Ther. 2007 Sep;15(9):1677-85. Epub 2007 Jun 12.


Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector.

Liu YL, Mingozzi F, Rodriguéz-Colôn SM, Joseph S, Dobrzynski E, Suzuki T, High KA, Herzog RW.

Hum Gene Ther. 2004 Aug;15(8):783-92.


Efficient induction of immune tolerance to coagulation factor IX following direct intramuscular gene transfer.

Cohn EF, Zhuo J, Kelly ME, Chao HJ.

J Thromb Haemost. 2007 Jun;5(6):1227-36.


Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer.

Mingozzi F, Liu YL, Dobrzynski E, Kaufhold A, Liu JH, Wang Y, Arruda VR, High KA, Herzog RW.

J Clin Invest. 2003 May;111(9):1347-56.


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

Fabb SA, Dickson JG.

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

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