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Refinado por: Nome da Publicação: Blood remover assunto: Dependovirus - Genetics 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
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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3
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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4
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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