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1
Novel approaches to hemophilia therapy: successes and challenges
Novel approaches to hemophilia therapy: successes and challenges
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Novel approaches to hemophilia therapy: successes and challenges

Arruda, Valder R. ; Doshi, Bhavya S. ; Samelson-Jones, Benjamin J.

Blood, 2017-11, Vol.130 (21), p.2251-2256 [Periódico revisado por pares]

United States: Elsevier Inc

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2
Assessing the potential for AAV vector genotoxicity in a murine model
Assessing the potential for AAV vector genotoxicity in a murine model
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Assessing the potential for AAV vector genotoxicity in a murine model

Li, Hojun ; Malani, Nirav ; Hamilton, Shari R. ; Schlachterman, Alexander ; Bussadori, Giulio ; Edmonson, Shyrie E. ; Shah, Rachel ; Arruda, Valder R. ; Mingozzi, Federico ; Fraser Wright, J. ; Bushman, Frederic D. ; High, Katherine A.

Blood, 2011-03, Vol.117 (12), p.3311-3319 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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3
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype
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Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Siner, Joshua I. ; Iacobelli, Nicholas P. ; Sabatino, Denise E. ; Ivanciu, Lacramiora ; Zhou, Shangzhen ; Poncz, Mortimer ; Camire, Rodney M. ; Arruda, Valder R.

Blood, 2013-05, Vol.121 (21), p.4396-4403 [Periódico revisado por pares]

United States: Elsevier Inc

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4
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy
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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy

Finn, Jonathan D. ; Ozelo, Margareth C. ; Sabatino, Denise E. ; Franck, Helen W.G. ; Merricks, Elizabeth P. ; Crudele, Julie M. ; Zhou, Shangzhen ; Kazazian, Haig H. ; Lillicrap, David ; Nichols, Timothy C. ; Arruda, Valder R.

Blood, 2010-12, Vol.116 (26), p.5842-5848 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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5
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver
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Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver

Mingozzi, Federico ; Hasbrouck, Nicole C. ; Basner-Tschakarjan, Etiena ; Edmonson, Shyrie A. ; Hui, Daniel J. ; Sabatino, Denise E. ; Zhou, Shangzhen ; Wright, J. Fraser ; Jiang, Haiyan ; Pierce, Glenn F. ; Arruda, Valder R. ; High, Katherine A.

Blood, 2007-10, Vol.110 (7), p.2334-2341 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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6
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B
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AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B

Manno, Catherine S. ; Chew, Amy J. ; Hutchison, Sylvia ; Larson, Peter J. ; Herzog, Roland W. ; Arruda, Valder R. ; Tai, Shing Jen ; Ragni, Margaret V. ; Thompson, Arthur ; Ozelo, Margareth ; Couto, Linda B. ; Leonard, Debra G.B. ; Johnson, Frederick A. ; McClelland, Alan ; Scallan, Ciaran ; Skarsgard, Erik ; Flake, Alan W. ; Kay, Mark A. ; High, Katherine A. ; Glader, Bertil

Blood, 2003-04, Vol.101 (8), p.2963-2972 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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7
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B
Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B
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Peripheral transvenular delivery of adeno-associated viral vectors to skeletal muscle as a novel therapy for hemophilia B

Arruda, Valder R. ; Stedman, Hansell H. ; Haurigot, Virginia ; Buchlis, George ; Baila, Stefano ; Favaro, Patricia ; Chen, Yifeng ; Franck, Helen G. ; Zhou, Shangzhen ; Wright, J. Fraser ; Couto, Linda B. ; Jiang, Haiyan ; Pierce, Glenn F. ; Bellinger, Dwight A. ; Mingozzi, Federico ; Nichols, Timothy C. ; High, Katherine A.

Blood, 2010-06, Vol.115 (23), p.4678-4688 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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8
Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response
Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response
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Omental implantation of BOECs in hemophilia dogs results in circulating FVIII antigen and a complex immune response

Ozelo, Margareth C. ; Vidal, Barbara ; Brown, Christine ; Notley, Colleen ; Hegadorn, Carol ; Webster, Sandra ; Harpell, Lori ; Ahlin, James ; Winterborn, Andrew ; Handforth, Janine ; Arruda, Valder R. ; Hough, Christine ; Lillicrap, David

Blood, 2014-06, Vol.123 (26), p.4045-4053 [Periódico revisado por pares]

United States: Elsevier Inc

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9
Immune complications and their management in inherited and acquired bleeding disorders
Immune complications and their management in inherited and acquired bleeding disorders
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Immune complications and their management in inherited and acquired bleeding disorders

Arruda, Valder R. ; Lillicrap, David ; Herzog, Roland W.

Blood, 2022-09, Vol.140 (10), p.1075-1085 [Periódico revisado por pares]

United States: Elsevier Inc

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10
Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo
Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo
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Factor IXa variants resistant to plasma inhibitors enhance clot formation in vivo

Ivanciu, Lacramioara ; Arruda, Valder R. ; Camire, Rodney M.

Blood, 2023-04, Vol.141 (16), p.2022-2032 [Periódico revisado por pares]

United States: Elsevier Inc

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