Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.
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Molecular therapy of primary hyperoxaluria.Gene Therapy with BMN 270 Results in Therapeutic Levels of FVIII in Mice and Primates and Normalization of Bleeding in Hemophilic Mice.Targeted in vivo knock-in of human alpha-1-antitrypsin cDNA using adenoviral delivery of CRISPR/Cas9.Platelet-Targeted Gene Therapy for Hemophilia.
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Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A.
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article científic
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article scientifique
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articolo scientifico
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artigo científico
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bilimsel makale
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scientific article published on 17 October 2016
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vedecký článok
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vetenskaplig artikel
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videnskabelig artikel
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Novel factor VIII variants wit ...... gene therapy for hemophilia A.
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Novel factor VIII variants wit ...... gene therapy for hemophilia A.
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Novel factor VIII variants wit ...... gene therapy for hemophilia A.
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Novel factor VIII variants wit ...... gene therapy for hemophilia A.
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Novel factor VIII variants wit ...... gene therapy for hemophilia A.
@en
Novel factor VIII variants wit ...... gene therapy for hemophilia A.
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Novel factor VIII variants wit ...... gene therapy for hemophilia A
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C B Zander
D E Sabatino
G N Nguyen
L A George
R J Davidson
R M Camire
V R Arruda
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P304
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10.1111/JTH.13543
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2016-11-25T00:00:00Z