Search Results - "McCarty, David A"
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Directed Engineering of a High-expression Chimeric Transgene as a Strategy for Gene Therapy of Hemophilia A
Published in Molecular therapy (01-07-2009)“…Human coagulation factor VIII (fVIII) is inefficiently biosynthesized in vitro and has proven difficult to express at therapeutic levels using available…”
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Comparison of factor VIII transgenes bioengineered for improved expression in gene therapy of hemophilia A
Published in Human gene therapy (01-05-2009)“…Successful gene therapy of hemophilia A depends on the sustained expression of therapeutic levels of factor VIII (fVIII). Because of mRNA instability,…”
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High-Expression Porcine FVIII Driven by Erythroid-Specific Promoters for Lentiviral Vector-Mediated Gene Therapy
Published in Blood (16-11-2008)“…Hemophilia A is an X-linked gene disorder that results in a deficiency of circulating coagulation factor VIII (fVIII) and may be ameliorated by only modest…”
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Journal Article