| Title |
Progress towards gene therapy for haemophilia B
|
|---|---|
| Published in |
International Journal of Hematology, February 2014
|
| DOI | 10.1007/s12185-014-1523-0 |
| Pubmed ID | |
| Authors |
Nishil Patel, Ulrike Reiss, Andrew M. Davidoff, Amit C. Nathwani |
| Abstract |
Haemophilia B is an X-linked recessive bleeding disorder, arising from a deficiency of coagulation factor IX. It has been a target for gene therapy ever since the factor IX gene was cloned in 1982. Several distinct approaches have been evaluated in humans over the last 30 years, but none has resulted in tangible corrections of the bleeding phenotype in humans until recently. Our group has now shown that lasting clinical improvement of the bleeding phenotype in patients with haemophilia B is possible following a single systemic administration of a self-complementary adeno-associated virus vector to deliver an optimised factor IX expression cassette to the liver. Success in this trial raises hope for patients with severe haemophilia B as well as others with inherited monogenetic disorders of the liver where current treatment options are limited. |
X Demographics
The data shown below were collected from the profile of 1 X user who shared this research output. Click here to find out more about how the information was compiled.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| France | 1 | 100% |
Demographic breakdown
| Type | Count | As % |
|---|---|---|
| Members of the public | 1 | 100% |
Mendeley readers
The data shown below were compiled from readership statistics for 47 Mendeley readers of this research output. Click here to see the associated Mendeley record.
Geographical breakdown
| Country | Count | As % |
|---|---|---|
| India | 1 | 2% |
| Iceland | 1 | 2% |
| Korea, Republic of | 1 | 2% |
| Unknown | 44 | 94% |
Demographic breakdown
| Readers by professional status | Count | As % |
|---|---|---|
| Researcher | 8 | 17% |
| Student > Master | 8 | 17% |
| Student > Bachelor | 8 | 17% |
| Student > Ph. D. Student | 7 | 15% |
| Librarian | 3 | 6% |
| Other | 7 | 15% |
| Unknown | 6 | 13% |
| Readers by discipline | Count | As % |
|---|---|---|
| Agricultural and Biological Sciences | 12 | 26% |
| Medicine and Dentistry | 11 | 23% |
| Biochemistry, Genetics and Molecular Biology | 10 | 21% |
| Pharmacology, Toxicology and Pharmaceutical Science | 2 | 4% |
| Psychology | 1 | 2% |
| Other | 3 | 6% |
| Unknown | 8 | 17% |
Attention Score in Context
This research output has an Altmetric Attention Score of 4. 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 03 March 2016.
All research outputs
#6,939,786
of 22,753,345 outputs
Outputs from International Journal of Hematology
#228
of 1,389 outputs
Outputs of similar age
#83,667
of 307,263 outputs
Outputs of similar age from International Journal of Hematology
#4
of 17 outputs
Altmetric has tracked 22,753,345 research outputs across all sources so far. This one has received more attention than most of these and is in the 68th percentile.
So far Altmetric has tracked 1,389 research outputs from this source. They receive a mean Attention Score of 3.6. This one has done well, scoring higher than 83% of its peers.
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 307,263 tracked outputs that were published within six weeks on either side of this one in any source. This one has gotten more attention than average, scoring higher than 71% of its contemporaries.
We're also able to compare this research output to 17 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 76% of its contemporaries.