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Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

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

  • In the top 5% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (99th percentile)
  • High Attention Score compared to outputs of the same age and source (88th percentile)

Mentioned by

news
15 news outlets
blogs
4 blogs
twitter
66 X users
patent
57 patents
facebook
3 Facebook pages
reddit
1 Redditor

Citations

dimensions_citation
740 Dimensions

Readers on

mendeley
1099 Mendeley
citeulike
3 CiteULike
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Title
Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo
Published in
Nature Biotechnology, February 2016
DOI 10.1038/nbt.3471
Pubmed ID
Authors

Hao Yin, Chun-Qing Song, Joseph R Dorkin, Lihua J Zhu, Yingxiang Li, Qiongqiong Wu, Angela Park, Junghoon Yang, Sneha Suresh, Aizhan Bizhanova, Ankit Gupta, Mehmet F Bolukbasi, Stephen Walsh, Roman L Bogorad, Guangping Gao, Zhiping Weng, Yizhou Dong, Victor Koteliansky, Scot A Wolfe, Robert Langer, Wen Xue, Daniel G Anderson

Abstract

The combination of Cas9, guide RNA and repair template DNA can induce precise gene editing and the correction of genetic diseases in adult mammals. However, clinical implementation of this technology requires safe and effective delivery of all of these components into the nuclei of the target tissue. Here, we combine lipid nanoparticle-mediated delivery of Cas9 mRNA with adeno-associated viruses encoding a sgRNA and a repair template to induce repair of a disease gene in adult animals. We applied our delivery strategy to a mouse model of human hereditary tyrosinemia and show that the treatment generated fumarylacetoacetate hydrolase (Fah)-positive hepatocytes by correcting the causative Fah-splicing mutation. Treatment rescued disease symptoms such as weight loss and liver damage. The efficiency of correction was >6% of hepatocytes after a single application, suggesting potential utility of Cas9-based therapeutic genome editing for a range of diseases.

X Demographics

X Demographics

The data shown below were collected from the profiles of 66 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 1,099 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
United States 8 <1%
Germany 3 <1%
Finland 2 <1%
Turkey 2 <1%
Spain 2 <1%
Netherlands 1 <1%
China 1 <1%
Canada 1 <1%
Belgium 1 <1%
Other 1 <1%
Unknown 1077 98%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 234 21%
Researcher 174 16%
Student > Bachelor 154 14%
Student > Master 122 11%
Student > Doctoral Student 55 5%
Other 145 13%
Unknown 215 20%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 328 30%
Agricultural and Biological Sciences 219 20%
Engineering 55 5%
Medicine and Dentistry 50 5%
Chemistry 42 4%
Other 152 14%
Unknown 253 23%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 190. 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 14 May 2024.
All research outputs
#215,971
of 25,954,278 outputs
Outputs from Nature Biotechnology
#442
of 8,655 outputs
Outputs of similar age
#3,716
of 408,975 outputs
Outputs of similar age from Nature Biotechnology
#13
of 118 outputs
Altmetric has tracked 25,954,278 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 99th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 8,655 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 44.6. This one has done particularly well, scoring higher than 94% 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 408,975 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 99% of its contemporaries.
We're also able to compare this research output to 118 others from the same source and published within six weeks on either side of this one. This one has done well, scoring higher than 88% of its contemporaries.