Merry Christmas for Patients with Hemophilia B

From the Departments of Internal Medicine and Biochemistry and Molecular Biophysics, Washington University School of Medicine, St. Louis.
New England Journal of Medicine (Impact Factor: 54.42). 12/2011; 365(25):2424-5. DOI: 10.1056/NEJMe1111138
Source: PubMed

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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    ABSTRACT: After clearing many hurdles, gene therapy is now reaching the commercialization stage and can be expected to make a modest but real contribution to treatment in the near future.
    Medecine sciences: M/S 10/2013; 29(10):923-5. DOI:10.1051/medsci/20132910023 · 0.52 Impact Factor
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    ABSTRACT: Background Haemophilia is a genetic disorder which is characterized by spontaneous or provoked, often uncontrolled, bleeding into joints, muscles and other soft tissues. Current methods of treatment are expensive, challenging and involve regular administration of clotting factors. Gene therapy has recently been prompted as a curative treatment modality. Objectives To evaluate the safety and efficacy of gene therapy for treating people with haemophilia A or B. Search methods We searched the Cochrane Cystic Fibrosis & Genetic Disorders Group's Coagulopathies Trials Register, compiled from electronic database searches and handsearching of journals and conference abstract books. We also searched the reference lists of relevant articles and reviews. Date of last search: 06 November 2014. Selection criteria Eligible trials included randomised or quasi-randomised clinical trials, including controlled clinical trials comparing gene therapy (with or without standard treatment) with standard treatment (factor replacement) or other 'curative' treatment such as stem cell transplantation individuals with haemophilia A or B of all ages who do not have inhibitors to factor VIII or IX. Data collection and analysis No trials of gene therapy for haemophilia were found. Main results No trials of gene therapy for haemophilia were identified. Authors' conclusions No randomised or quasi-randomised clinical trials of gene therapy for haemophilia were identified. Thus, we are unable to determine the effects of gene therapy for haemophilia. Gene therapy for haemophilia is still in its nascent stages and there is a need for well-designed clinical trials to assess the long-term feasibility, success and risks of gene therapy for people with haemophilia.
    Cochrane database of systematic reviews (Online) 11/2014; 11(11):CD010822. DOI:10.1002/14651858.CD010822.pub2 · 5.70 Impact Factor
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    08/2012; Suppl 1. DOI:10.4172/2157-7412.S1-016


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