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Developments in the treatment of hemophilia B: focus on emerging gene therapy

Overview of attention for article published in The Application of Clinical Genetics, October 2013
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Title
Developments in the treatment of hemophilia B: focus on emerging gene therapy
Published in
The Application of Clinical Genetics, October 2013
DOI 10.2147/tacg.s31928
Pubmed ID
Authors

John Gray, Maria Cancio, Ulrike Reiss, Amit Nathwani, Andrew Davidoff

Abstract

Hemophilia B is a genetic disorder that is characterized by a deficiency of clotting factor IX (FIX) and excessive bleeding. Advanced understanding of the pathophysiology of the disease has led to the development of improved treatment strategies that aim to minimize the acute and long-term complications of the disease. Patients with hemophilia B are ideal candidates for gene therapy, mostly because a small increase in protein production can lead to significantly decreased bleeding diathesis. Although human clotting FIX was cloned and sequenced over 30 years ago, progress toward achieving real success in human clinical trials has been slow, with long-term, therapeutically relevant gene expression only achieved in one trial published in 2011. The history of this extensive research effort has revealed the importance of the interactions between gene therapy vectors and multiple arms of the host immune system at multiple stages of the transduction process. Different viral vector systems each have unique properties that influence their ability to deliver genes to different tissues, and the data generated in several clinical trials testing different vectors for hemophilia have guided our understanding toward development of optimal configurations for treating hemophilia B. The recent clinical success implementing a novel adeno-associated virus vector demonstrated sufficient FIX expression in patients to convert a severe hemophilia phenotype to mild, an achievement which has the potential to profoundly alter the impact of this disease on human society. Continued research should lead to vector designs that result in higher FIX activity at lower vector doses and with reduced host immune responses to the vector and the transgene product.

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Mendeley readers

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Geographical breakdown

Country Count As %
Iceland 1 1%
South Africa 1 1%
Canada 1 1%
Brazil 1 1%
Unknown 67 94%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 17 24%
Student > Ph. D. Student 15 21%
Researcher 11 15%
Student > Master 10 14%
Other 3 4%
Other 8 11%
Unknown 7 10%
Readers by discipline Count As %
Agricultural and Biological Sciences 20 28%
Biochemistry, Genetics and Molecular Biology 16 23%
Medicine and Dentistry 16 23%
Pharmacology, Toxicology and Pharmaceutical Science 3 4%
Nursing and Health Professions 1 1%
Other 7 10%
Unknown 8 11%