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A correction has been published: N Engl J Med 2001;345(5):384.

Review Article
Medical Progress
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Volume 344:1773-1779 June 7, 2001 Number 23
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The Hemophilias — From Royal Genes to Gene Therapy
Pier M. Mannucci, M.D., and Edward G.D. Tuddenham, M.D.

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Of the various types of hemophilia, the most common of these lifelong bleeding disorders are due to an inherited deficiency of factor VIII or factor IX (Table 1). The genes for these blood coagulation factors lie on the X chromosome, and when mutated, they cause the X-linked recessive traits hemophilia A and B. Since these disorders are X-linked, they usually occur in males. Usually, the affected boy has inherited the mutant gene (XH) from his carrier mother (XH/X ), but about 30 percent of cases arise from a spontaneous mutation, and there is . . . [Full Text of this Article]

Effects of DNA and Biochemical Techniques

The Choice of Replacement Therapy

Plasma-Derived Factors

Recombinant Products

Selecting the Appropriate Product

Treatment of Patients Who Have Inhibitors

Secondary Diseases in Patients with Hemophilia

Gene Therapy

Conclusions


Source Information

From the Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, Istituto di Ricovero e Cura a Carattere Scientifico Maggiore Hospital and University of Milan, Milan, Italy (P.M.M.); and the Medical Research Council Clinical Sciences Centre, Imperial College School of Medicine, Hammersmith Hospital, London (E.G.D.T.).

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