The critical need for postmarketing surveillance in gene therapy for haemophilia
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چکیده
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15 صفحه اولGene therapy for haemophilia
The ultimate goal of gene therapy is the replacement of a defective gene sequence with a corrected version to eliminate disease for the lifetime of the patient. This challenging task is not yet accomplished, however significant progress is evident. An initial spate of clinical trials attempting the treatment of haemophilia with gene transfer primarily resulted in the demonstration of good safet...
متن کاملGene therapy for haemophilia
Haemophilia is a congenital coagulation disorder characterized by uncontrolled haemorrhagic episodes that are crippling and potentially life-threatening. Haemophilia A results from subnormal levels of an essential cofactor protein, factor VIII (F.VIII), and affects 1 in every 10,000 males; haemophilia B is associated with a lack of an essential protease, factor IX (F.IX), and occurs in 1 out of...
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15 صفحه اولGene Therapy in Haemophilia
Haemophilia A (factor VIII {FVIII} deficiency) and haemophilia B (factor IX {FIX} deficiency) are rare X-linked bleeding disorders occurring at an incidence of 1:5,000 and 1:25,000 males throughout the world. Treatment of these conditions by replacement therapy with plasma-derived FVIII or FIX concentrates has been established in the developed world since the mid 1970s, but has brought with it ...
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ژورنال
عنوان ژورنال: Haemophilia
سال: 2020
ISSN: 1351-8216,1365-2516
DOI: 10.1111/hae.13972