Unique strategies for therapeutic gene transfer in haemophilia A and haemophilia B
نویسندگان
چکیده
منابع مشابه
Haemophilia A and haemophilia B: molecular insights.
This review focuses on selected areas that should interest both the scientist and the clinician alike: polymorphisms within the factor VIII and factor IX genes, their linkage, and their ethnic variation; a general assessment of mutations within both genes and a detailed inspection of the molecular pathology of certain mutations to illustrate the diverse cause-effect relations that exist; a summ...
متن کاملProgress towards gene therapy for haemophilia B.
Haemophilia B is an X-linked recessive bleeding disorder, arising from a deficiency of coagulation factor IX. It has been a target for gene therapy ever since the factor IX gene was cloned in 1982. Several distinct approaches have been evaluated in humans over the last 30 years, but none has resulted in tangible corrections of the bleeding phenotype in humans until recently. Our group has now s...
متن کاملGene 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...
متن کاملGene 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: 2010
ISSN: 1351-8216,1365-2516
DOI: 10.1111/j.1365-2516.2010.02290.x