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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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  • Good Attention Score compared to outputs of the same age (75th percentile)

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2 X users
7 patents
1 Facebook page


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73 Mendeley
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

Maria I Cancio, Ulrike M Reiss, Amit C Nathwani, Andrew M Davidoff, John T Gray


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.

X Demographics

X Demographics

The data shown below were collected from the profiles of 2 X users who shared this research output. Click here to find out more about how the information was compiled.
Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 73 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Iceland 1 1%
Canada 1 1%
Brazil 1 1%
Unknown 70 96%

Demographic breakdown

Readers by professional status Count As %
Student > Bachelor 17 23%
Student > Ph. D. Student 14 19%
Researcher 11 15%
Student > Master 10 14%
Other 4 5%
Other 9 12%
Unknown 8 11%
Readers by discipline Count As %
Agricultural and Biological Sciences 19 26%
Biochemistry, Genetics and Molecular Biology 16 22%
Medicine and Dentistry 16 22%
Pharmacology, Toxicology and Pharmaceutical Science 4 5%
Nursing and Health Professions 1 1%
Other 7 10%
Unknown 10 14%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 6. This is our high-level measure of the quality and quantity of online attention that it has received. This Attention Score, as well as the ranking and number of research outputs shown below, was calculated when the research output was last mentioned on 13 February 2024.
All research outputs
of 25,748,735 outputs
Outputs from The Application of Clinical Genetics
of 1 outputs
Outputs of similar age
of 220,793 outputs
Outputs of similar age from The Application of Clinical Genetics
of 1 outputs
Altmetric has tracked 25,748,735 research outputs across all sources so far. This one has received more attention than most of these and is in the 74th percentile.
So far Altmetric has tracked 1 research outputs from this source. They typically receive a little more attention than average, with a mean Attention Score of 5.2. This one scored the same or higher as 0 of them.
Older research outputs will score higher simply because they've had more time to accumulate mentions. To account for age we can compare this Altmetric Attention Score to the 220,793 tracked outputs that were published within six weeks on either side of this one in any source. This one has done well, scoring higher than 75% of its contemporaries.
We're also able to compare this research output to 1 others from the same source and published within six weeks on either side of this one. This one has scored higher than all of them