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Refinado por: Nome da Publicação: Blood remover assunto: Hemophilia B - Therapy remover
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1
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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2
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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3
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
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
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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, Valder R. ; Schuettrumpf, Joerg ; Herzog, Roland W. ; Nichols, Timothy C. ; Robinson, Nancy ; Lotfi, Yasmin ; Mingozzi, Federico ; Xiao, Weidong ; Couto, Linda B. ; High, Katherine A.

Blood, 2004-01, Vol.103 (1), p.85-92 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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4
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice
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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Crudele, Julie M. ; Finn, Jonathan D. ; Siner, Joshua I. ; Martin, Nicholas B. ; Niemeyer, Glenn P. ; Zhou, Shangzhen ; Mingozzi, Federico ; Lothrop, Clinton D. ; Arruda, Valder R.

Blood, 2015-03, Vol.125 (10), p.1553-1561 [Periódico revisado por pares]

United States: Elsevier Inc

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5
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model
Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model
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Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

Arruda, Valder R. ; Stedman, Hansell H. ; Nichols, Timothy C. ; Haskins, Mark E. ; Nicholson, Matthew ; Herzog, Roland W. ; Couto, Linda B. ; High, Katherine A.

Blood, 2005-05, Vol.105 (9), p.3458-3464 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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6
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy
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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

Mount, Jane D. ; Herzog, Roland W. ; Tillson, D. Michael ; Goodman, Susan A. ; Robinson, Nancy ; McCleland, Mark L. ; Bellinger, Dwight ; Nichols, Timothy C. ; Arruda, Valder R. ; Lothrop, Clinton D. ; High, Katherine A.

Blood, 2002-04, Vol.99 (8), p.2670-2676 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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7
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy
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Long-term correction of inhibitor-prone hemophilia B dogs treated with liver-directed AAV2-mediated factor IX gene therapy

Niemeyer, Glenn P. ; Herzog, Roland W. ; Mount, Jane ; Arruda, Valder R. ; Tillson, D. Michael ; Hathcock, John ; van Ginkel, Frederik W. ; High, Katherine A. ; Lothrop, Clinton D.

Blood, 2009-01, Vol.113 (4), p.797-806 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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8
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy
The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy
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The efficacy and the risk of immunogenicity of FIX Padua (R338L) in hemophilia B dogs treated by AAV muscle gene therapy

Finn, Jonathan D. ; Nichols, Timothy C. ; Svoronos, Nikolaos ; Merricks, Elizabeth P. ; Bellenger, Dwight A. ; Zhou, Shangshen ; Simioni, Paolo ; High, Katherine A. ; Arruda, Valder R.

Blood, 2012-11, Vol.120 (23), p.4521-4523 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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9
Factor IX variants improve gene therapy efficacy for hemophilia B
Factor IX variants improve gene therapy efficacy for hemophilia B
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Factor IX variants improve gene therapy efficacy for hemophilia B

Schuettrumpf, Joerg ; Herzog, Roland W. ; Schlachterman, Alexander ; Kaufhold, Antje ; Stafford, Darrel W. ; Arruda, Valder R.

Blood, 2005-03, Vol.105 (6), p.2316-2323 [Periódico revisado por pares]

Washington, DC: Elsevier Inc

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10
The liver sieve and gene therapy
The liver sieve and gene therapy
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The liver sieve and gene therapy

Fraser, Robin ; Le Couteur, David G. ; Warren, Alessandra ; Cogger, Victoria C. ; Bertolino, Patrick ; Smith, Mark

Blood, 2003-04, Vol.101 (8), p.3338-3338 [Periódico revisado por pares]

United States: Elsevier Inc

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