Curing hemophilia A by NHEJ-mediated ectopic F8 insertion in the mouse
نویسندگان
چکیده
منابع مشابه
Gene Expression under F8 Promoter Driving In Mouse Hepatoma Cells: A Step towards Gene Therapy of Hemophilia
Background and Objectives: Significant progress has been made in treatment of hemophilia. Ex-vivo gene therapy is going popular due to the capability of this method in using isogenic cells for genetic manipulation and reintroducing them into same host after proliferation. Most gene therapy techniques use viral vectors, which usually harbor a strong and non-specific promoter (e...
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Hemophilia, a common congenital coagulation disorder, is classified as hemophilia A (HA) and hemophilia B (HB), which result from a deficiency or dysfunction of coagulation factor VIII (FVIII) and factor IX (FIX), respectively. HA is known to be caused by heterogeneous mutations of the FVIII gene (F8), such as inversions, substitutions, deletions, insertions, etc. F8 (NM_000132.3) is located on...
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UNLABELLED ESSENTIALS: Anti-factor VIII (FVIII) inhibitory antibody formation is a severe complication in hemophilia A therapy. We genetically engineered and characterized a mouse model with complete deletion of the F8 coding region. F8(TKO) mice exhibit severe hemophilia, express no detectable F8 mRNA, and produce FVIII inhibitors. The defined background and lack of FVIII in F8(TKO) mice will ...
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Although uncommon, acquired hemophilia A (HA) is associated with a high rate of mortality due to severe bleeding. In spite of many hypotheses regarding the cause of acquired HA, there is as yet no established theory. In this study, we investigated the possibility that mutation(s) in the F8 gene may be correlated with the development of inhibitory autoantibodies. Direct sequencing analysis was p...
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ژورنال
عنوان ژورنال: Genome Biology
سال: 2019
ISSN: 1474-760X
DOI: 10.1186/s13059-019-1907-9