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Items: 1 to 20 of 128

1.

Safety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.

Haurigot V, Mingozzi F, Buchlis G, Hui DJ, Chen Y, Basner-Tschakarjan E, Arruda VR, Radu A, Franck HG, Wright JF, Zhou S, Stedman HH, Bellinger DA, Nichols TC, High KA.

Mol Ther. 2010 Jul;18(7):1318-29. doi: 10.1038/mt.2010.73. Epub 2010 Apr 27.

2.

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.

Blood. 2010 Jun 10;115(23):4678-88. doi: 10.1182/blood-2009-12-261156. Epub 2010 Mar 24.

3.

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.

PMID:
12162811
4.

Persistent expression of canine factor IX in hemophilia B canines.

Chao H, Samulski R, Bellinger D, Monahan P, Nichols T, Walsh C.

Gene Ther. 1999 Oct;6(10):1695-704.

5.

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.

6.

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.

7.

Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer.

Buchlis G, Podsakoff GM, Radu A, Hawk SM, Flake AW, Mingozzi F, High KA.

Blood. 2012 Mar 29;119(13):3038-41. doi: 10.1182/blood-2011-09-382317. Epub 2012 Jan 23. Erratum in: Blood. 2014 Mar 13;123(11):1768.

8.

AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects.

Hasbrouck NC, High KA.

Gene Ther. 2008 Jun;15(11):870-5. doi: 10.1038/gt.2008.71. Epub 2008 Apr 24. Review.

PMID:
18432276
9.

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.

PMID:
17594244
10.

Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver.

Wang L, Nichols TC, Read MS, Bellinger DA, Verma IM.

Mol Ther. 2000 Feb;1(2):154-8.

11.

AAV-mediated gene transfer for hemophilia.

High KA.

Ann N Y Acad Sci. 2001 Dec;953:64-74. Review.

PMID:
11795424
12.

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia.

Monahan PE, Samulski RJ, Tazelaar J, Xiao X, Nichols TC, Bellinger DA, Read MS, Walsh CE.

Gene Ther. 1998 Jan;5(1):40-9.

13.

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.

14.

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.

PMID:
15889137
15.

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.

PMID:
15319035
16.

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy.

Wang L, Calcedo R, Nichols TC, Bellinger DA, Dillow A, Verma IM, Wilson JM.

Blood. 2005 Apr 15;105(8):3079-86. Epub 2005 Jan 6.

17.

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.

18.

Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: preclinical evaluation supporting an ongoing adeno-associated virus clinical trial.

Monahan PE, Sun J, Gui T, Hu G, Hannah WB, Wichlan DG, Wu Z, Grieger JC, Li C, Suwanmanee T, Stafford DW, Booth CJ, Samulski JJ, Kafri T, McPhee SW, Samulski RJ.

Hum Gene Ther. 2015 Feb;26(2):69-81. doi: 10.1089/hum.2014.106. Epub 2015 Jan 21.

19.

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.

PMID:
10700178
20.

Muscle-directed gene therapy for hemophilia B with more efficient and less immunogenic AAV vectors.

Wang L, Louboutin JP, Bell P, Greig JA, Li Y, Wu D, Wilson JM.

J Thromb Haemost. 2011 Oct;9(10):2009-19. doi: 10.1111/j.1538-7836.2011.04491.x.

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