AAV5–Factor VIII Gene Transfer in Severe Hemophilia A
نویسندگان
چکیده
منابع مشابه
Factor VIII products and inhibitors in severe hemophilia A.
n engl j med 368;15 nejm.org april 11, 2013 1456 improves outcomes as compared with less complete revascularization.2 However, our trial involved highly experienced interventional cardiologists and surgeons who attempted to maximize the completeness of revascularization. Substantially more complete PCI-based revascularization is therefore unlikely in other institutions. Serruys and Farooq sugge...
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Hemophilia A, the commonest form of hemophilia, affects 1 in 5,000 males in the general population. This X-linked disease is often associated with mutations spread along the factor Vll l gene.' In a number of cases, no point mutations have been detected, but a strikingly abnormal mRNA transcript has been shown using Northem blot analysis. It has subsequently been found that large DNA inversions...
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Twenty-two molecular diagnostic laboratories from 14 countries participated in a consortium study to estimate the impact of Factor VIII gene inversions in severe hemophilia A. A total of 2,093 patients with severe hemophilia A were studied; of those, 740 (35%) had a type 1 (distal) factor VIII inversion, and 140 (7%) showed a type 2 (proximal) inversion. In 25 cases, the molecular analysis show...
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Background: Factor VIII administration to hemophilia A patients results in an immune response (inhibitor formation) which significantly complicates the therapy. The present study was performed to determine the prevalence of inhibitor development in hemophilia A patients receiving recombinant factor VIII therapy. Materials and Methods: This was an observational descriptive study. Clotting fac...
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H EMOPI-IILIA A, the most common congenital bleeding disorder, affects I in 10,000 to 20,000 males. The molecular lesions are heterogeneous and only I 0% of specific defects had been characterized so far. These included ddetions and point mutations.’ 8 Most of the point mutations detected involved the recognition site (TCGA) for the restriction enzyme Taq I and of these, most are associated wit...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2017
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa1708483