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Update on Progress and Hurdles in Novel Genetic Therapies for Hemophilia

1 Jan 2007

Abstract excerpt

Among inherited disorders, hemophilia has a number of characteristics that make it attractive as a model for gene transfer approaches. Several trials of gene therapy for hemophilia were carried out earlier in this decade; these trials were all first-in-class, i.e. the first use of a particular vector system in a particular target tissue, and thus yielded important safety data for the approaches under...

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PMID: 18024666
DOI: 10.1182/asheducation-2007.1.466

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