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Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease

Overview of attention for article published in Nature Biotechnology, March 2016
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About this Attention Score

  • In the top 25% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (92nd percentile)
  • Above-average Attention Score compared to outputs of the same age and source (60th percentile)

Mentioned by

blogs
1 blog
twitter
17 X users
patent
6 patents
facebook
1 Facebook page
f1000
1 research highlight platform

Citations

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167 Dimensions

Readers on

mendeley
173 Mendeley
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Title
Targeted gene addition in human CD34+ hematopoietic cells for correction of X-linked chronic granulomatous disease
Published in
Nature Biotechnology, March 2016
DOI 10.1038/nbt.3513
Pubmed ID
Authors

Suk See De Ravin, Andreas Reik, Pei-Qi Liu, Linhong Li, Xiaolin Wu, Ling Su, Castle Raley, Narda Theobald, Uimook Choi, Alexander H Song, Andy Chan, Jocelynn R Pearl, David E Paschon, Janet Lee, Hannah Newcombe, Sherry Koontz, Colin Sweeney, David A Shivak, Kol A Zarember, Madhusudan V Peshwa, Philip D Gregory, Fyodor D Urnov, Harry L Malech

Abstract

Gene therapy with genetically modified human CD34(+) hematopoietic stem and progenitor cells (HSPCs) may be safer using targeted integration (TI) of transgenes into a genomic 'safe harbor' site rather than random viral integration. We demonstrate that temporally optimized delivery of zinc finger nuclease mRNA via electroporation and adeno-associated virus (AAV) 6 delivery of donor constructs in human HSPCs approaches clinically relevant levels of TI into the AAVS1 safe harbor locus. Up to 58% Venus(+) HSPCs with 6-16% human cell marking were observed following engraftment into mice. In HSPCs from patients with X-linked chronic granulomatous disease (X-CGD), caused by mutations in the gp91phox subunit of the NADPH oxidase, TI of a gp91phox transgene into AAVS1 resulted in ∼15% gp91phox expression and increased NADPH oxidase activity in ex vivo-derived neutrophils. In mice transplanted with corrected HSPCs, 4-11% of human cells in the bone marrow expressed gp91phox. This method for TI into AAVS1 may be broadly applicable to correction of other monogenic diseases.

X Demographics

X Demographics

The data shown below were collected from the profiles of 17 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 173 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Finland 1 <1%
Turkey 1 <1%
Unknown 171 99%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 45 26%
Researcher 41 24%
Student > Bachelor 17 10%
Student > Master 12 7%
Other 10 6%
Other 18 10%
Unknown 30 17%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 68 39%
Agricultural and Biological Sciences 33 19%
Medicine and Dentistry 15 9%
Immunology and Microbiology 7 4%
Engineering 4 2%
Other 12 7%
Unknown 34 20%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 27. 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 30 August 2023.
All research outputs
#1,413,842
of 25,375,376 outputs
Outputs from Nature Biotechnology
#2,320
of 8,790 outputs
Outputs of similar age
#22,902
of 306,044 outputs
Outputs of similar age from Nature Biotechnology
#43
of 107 outputs
Altmetric has tracked 25,375,376 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 94th percentile: it's in the top 10% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 8,790 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 43.1. This one has gotten more attention than average, scoring higher than 73% 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 306,044 tracked outputs that were published within six weeks on either side of this one in any source. This one has done particularly well, scoring higher than 92% of its contemporaries.
We're also able to compare this research output to 107 others from the same source and published within six weeks on either side of this one. This one has gotten more attention than average, scoring higher than 60% of its contemporaries.