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2011•New England Journal of MedicineRequires access

Merry Christmas for Patients with Hemophilia B

Katherine Parker Ponder

Open publisher page 23 citations

Abstract

Hemophilia B (also known as Christmas disease) is due to deficiency of coagulation factor IX (FIX). In this issue of the Journal, Nathwani et al. report the first unequivocal evidence of successful gene therapy for hemophilia B — a major advance in this field.1 This success for hemophilia may translate into gene therapy for other blood protein deficiencies.Hemophilia is due to deficiency in a coagulation factor and results in a bleeding disorder that often involves joints and muscles. The most common types are hemophilia A and B, which are due to deficiencies of factor VIII and FIX, respectively, and . . .

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What this paper is about

Hemophilia B (also known as Christmas disease) is due to deficiency of coagulation factor IX (FIX). In this issue of the Journal, Nathwani et al. report the first unequivocal evidence of successful gene therapy for hemophilia B — a major advance in this field.1 This success for hemophilia may translate into gene therapy for other blood protein deficiencies.Hemophilia is due to deficiency in a coagulation factor and results in a bleeding disorder that often involves joints and muscles. The most common types are hemophilia A and B, which are due to deficiencies of factor VIII and FIX, respectively, and . . .

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Available abstract

Hemophilia B (also known as Christmas disease) is due to deficiency of coagulation factor IX (FIX). In this issue of the Journal, Nathwani et al. report the first unequivocal evidence of successful gene therapy for hemophilia B — a major advance in this field.1 This success for hemophilia may translate into gene therapy for other blood protein deficiencies.Hemophilia is due to deficiency in a coagulation factor and results in a bleeding disorder that often involves joints and muscles. The most common types are hemophilia A and B, which are due to deficiencies of factor VIII and FIX, respectively, and . . .

Key concepts: Medicine, Factor IX, Coagulation, Genetic enhancement, Coagulopathy, Coagulation Disorder, Blood coagulation factors, Disease

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