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Results: 1 to 20 of 181

1.

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.

PMID:
12969984
[PubMed - indexed for MEDLINE]
Free Article
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.

PMID:
12813929
[PubMed - indexed for MEDLINE]
Free PMC Article
3.

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.

PMID:
12515715
[PubMed - indexed for MEDLINE]
Free Article
4.

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.

PMID:
17362228
[PubMed - indexed for MEDLINE]
5.

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
[PubMed - indexed for MEDLINE]
6.

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
[PubMed - indexed for MEDLINE]
7.

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
[PubMed - indexed for MEDLINE]
8.

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
[PubMed - indexed for MEDLINE]
9.

Lack of germline transmission of vector sequences following systemic administration of recombinant AAV-2 vector in males.

Arruda VR, Fields PA, Milner R, Wainwright L, De Miguel MP, Donovan PJ, Herzog RW, Nichols TC, Biegel JA, Razavi M, Dake M, Huff D, Flake AW, Couto L, Kay MA, High KA.

Mol Ther. 2001 Dec;4(6):586-92.

PMID:
11735343
[PubMed - indexed for MEDLINE]
Free Article
10.

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
[PubMed - indexed for MEDLINE]
11.

AAV-mediated gene transfer for hemophilia.

High KA.

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

PMID:
11795424
[PubMed - indexed for MEDLINE]
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.

PMID:
9536263
[PubMed - indexed for MEDLINE]
Free Article
13.

Improved hepatic gene transfer by using an adeno-associated virus serotype 5 vector.

Mingozzi F, Schüttrumpf J, Arruda VR, Liu Y, Liu YL, High KA, Xiao W, Herzog RW.

J Virol. 2002 Oct;76(20):10497-502.

PMID:
12239326
[PubMed - indexed for MEDLINE]
Free PMC Article
14.

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.

PMID:
24460861
[PubMed - indexed for MEDLINE]
Free PMC Article
15.

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation.

Herzog RW, Mount JD, Arruda VR, High KA, Lothrop CD Jr.

Mol Ther. 2001 Sep;4(3):192-200.

PMID:
11545609
[PubMed - indexed for MEDLINE]
Free Article
16.

Efficacy and safety of adeno-associated viral vectors based on serotype 8 and 9 vs. lentiviral vectors for hemophilia B gene therapy.

Vandendriessche T, Thorrez L, Acosta-Sanchez A, Petrus I, Wang L, Ma L, DE Waele L, Iwasaki Y, Gillijns V, Wilson JM, Collen D, Chuah MK.

J Thromb Haemost. 2007 Jan;5(1):16-24. Epub 2006 Sep 26.

PMID:
17002653
[PubMed - indexed for MEDLINE]
17.

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.

PMID:
20335222
[PubMed - indexed for MEDLINE]
Free PMC Article
18.

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.

PMID:
19309290
[PubMed - indexed for MEDLINE]
Free PMC Article
19.

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.

PMID:
22565846
[PubMed - indexed for MEDLINE]
Free PMC Article
20.

AAV-mediated gene transfer to skeletal muscle.

Herzog RW.

Methods Mol Biol. 2004;246:179-94.

PMID:
14970592
[PubMed - indexed for MEDLINE]

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