Arruda VR - Search Results

1

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant.

George LA, Sullivan SK, Giermasz A, Rasko JEJ, Samelson-Jones BJ, Ducore J, Cuker A, Sullivan LM, Majumdar S, Teitel J, McGuinn CE, Ragni MV, Luk AY, Hui D, Wright JF, Chen Y, Liu Y, Wachtel K, Winters A, Tiefenbacher S, Arruda VR, van der Loo JCM, Zelenaia O, Takefman D, Carr ME, Couto LB, Anguela XM, High KA. George LA, et al. Among authors: arruda vr. N Engl J Med. 2017 Dec 7;377(23):2215-2227. doi: 10.1056/NEJMoa1708538. N Engl J Med. 2017. PMID: 29211678 Free PMC article. Clinical Trial.

2

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. Kay MA, et al. Nat Genet. 2000 Mar;24(3):257-61. doi: 10.1038/73464. Nat Genet. 2000. PMID: 10700178 Clinical Trial.

3

Regulation of human coagulation factor X gene expression by GATA-4 and the Sp family of transcription factors.

Hung HL, Pollak ES, Kudaravalli RD, Arruda V, Chu K, High KA. Hung HL, et al. Blood. 2001 Feb 15;97(4):946-51. doi: 10.1182/blood.v97.4.946. Blood. 2001. PMID: 11159521 Free article.

4

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. Mount JD, et al. Among authors: arruda vr. Blood. 2002 Apr 15;99(8):2670-6. doi: 10.1182/blood.v99.8.2670. Blood. 2002. PMID: 11929752 Free article.

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. Herzog RW, et al. Among authors: arruda vr. Hum Gene Ther. 2002 Jul 20;13(11):1281-91. doi: 10.1089/104303402760128513. Hum Gene Ther. 2002. PMID: 12162811

6

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. Mingozzi F, et al. Among authors: arruda vr. J Virol. 2002 Oct;76(20):10497-502. doi: 10.1128/jvi.76.20.10497-10502.2002. J Virol. 2002. PMID: 12239326 Free PMC article.

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. Manno CS, et al. Among authors: arruda vr. Blood. 2003 Apr 15;101(8):2963-72. doi: 10.1182/blood-2002-10-3296. Epub 2002 Dec 19. Blood. 2003. PMID: 12515715 Free article.

8

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. Mingozzi F, et al. Among authors: arruda vr. J Clin Invest. 2003 May;111(9):1347-56. doi: 10.1172/JCI16887. J Clin Invest. 2003. PMID: 12727926 Free PMC article.

9

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. Arruda VR, et al. Blood. 2004 Jan 1;103(1):85-92. doi: 10.1182/blood-2003-05-1446. Epub 2003 Sep 11. Blood. 2004. PMID: 12969984 Free PMC article.

10

Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII.

Margaritis P, Arruda VR, Aljamali M, Camire RM, Schlachterman A, High KA. Margaritis P, et al. Among authors: arruda vr. J Clin Invest. 2004 Apr;113(7):1025-31. doi: 10.1172/JCI20106. J Clin Invest. 2004. PMID: 15057309 Free PMC article.

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