The critical need for postmarketing surveillance in gene therapy for haemophilia

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Gene 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...

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Gene 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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Gene 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