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Items: 1 to 20 of 104

1.

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model.

Arruda VR, Stedman HH, Nichols TC, Haskins ME, Nicholson M, Herzog RW, Couto LB, High KA.

Blood. 2005 May 1;105(9):3458-64. Epub 2004 Oct 12.

2.

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.

3.

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.

4.

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
5.

Adeno-associated virus-mediated gene transfer for hemophilia B.

High KA.

Int J Hematol. 2002 Nov;76(4):310-8. Review.

PMID:
12463593
6.

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.

7.

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.

8.

AAV-mediated gene transfer for hemophilia.

High KA.

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

PMID:
11795424
9.

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.

10.

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.

11.
12.

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
13.

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.

14.

Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy.

Mount JD, Herzog RW, Tillson DM, Goodman SA, Robinson N, McCleland ML, Bellinger D, Nichols TC, Arruda VR, Lothrop CD Jr, High KA.

Blood. 2002 Apr 15;99(8):2670-6.

15.

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
16.

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.

17.

Clinical gene transfer studies for hemophilia B.

High KA.

Semin Thromb Hemost. 2004 Apr;30(2):257-67.

PMID:
15118937
18.

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.

19.

Intraarticular factor IX protein or gene replacement protects against development of hemophilic synovitis in the absence of circulating factor IX.

Sun J, Hakobyan N, Valentino LA, Feldman BL, Samulski RJ, Monahan PE.

Blood. 2008 Dec 1;112(12):4532-41. doi: 10.1182/blood-2008-01-131417. Epub 2008 Aug 20.

20.

AAV-mediated gene transfer for hemophilia.

High K.

Genet Med. 2002 Nov-Dec;4(6 Suppl):56S-61S. Review.

PMID:
12544490

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