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All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo

Overview of attention for article published in Genome Biology, September 2018
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  • In the top 5% of all research outputs scored by Altmetric
  • High Attention Score compared to outputs of the same age (92nd percentile)
  • Good Attention Score compared to outputs of the same age and source (70th percentile)

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2 blogs
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21 X users
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3 patents

Citations

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Title
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo
Published in
Genome Biology, September 2018
DOI 10.1186/s13059-018-1515-0
Pubmed ID
Authors

Raed Ibraheim, Chun-Qing Song, Aamir Mir, Nadia Amrani, Wen Xue, Erik J. Sontheimer

Abstract

Clustered, regularly interspaced, short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) have recently opened a new avenue for gene therapy. Cas9 nuclease guided by a single-guide RNA (sgRNA) has been extensively used for genome editing. Currently, three Cas9 orthologs have been adapted for in vivo genome engineering applications: Streptococcus pyogenes Cas9 (SpyCas9), Staphylococcus aureus Cas9 (SauCas9), and Campylobacter jejuni (CjeCas9). However, additional in vivo editing platforms are needed, in part to enable a greater range of sequences to be accessed via viral vectors, especially those in which Cas9 and sgRNA are combined into a single vector genome. Here, we present in vivo editing using Neisseria meningitidis Cas9 (NmeCas9). NmeCas9 is compact, edits with high accuracy, and possesses a distinct protospacer adjacent motif (PAM), making it an excellent candidate for safe gene therapy applications. We find that NmeCas9 can be used to target the Pcsk9 and Hpd genes in mice. Using tail-vein hydrodynamic-based delivery of NmeCas9 plasmid to target the Hpd gene, we successfully reprogram the tyrosine degradation pathway in Hereditary Tyrosinemia Type I mice. More importantly, we deliver NmeCas9 with its sgRNA in a single recombinant adeno-associated vector (rAAV) to target Pcsk9, resulting in lower cholesterol levels in mice. This all-in-one vector yielded > 35% gene modification after two weeks of vector administration, with minimal off-target cleavage in vivo. Our findings indicate that NmeCas9 can enable the editing of disease-causing loci in vivo, expanding the targeting scope of RNA-guided nucleases.

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X Demographics

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Mendeley readers

Mendeley readers

The data shown below were compiled from readership statistics for 117 Mendeley readers of this research output. Click here to see the associated Mendeley record.

Geographical breakdown

Country Count As %
Unknown 117 100%

Demographic breakdown

Readers by professional status Count As %
Student > Ph. D. Student 21 18%
Researcher 19 16%
Student > Bachelor 15 13%
Student > Master 12 10%
Other 5 4%
Other 15 13%
Unknown 30 26%
Readers by discipline Count As %
Biochemistry, Genetics and Molecular Biology 49 42%
Agricultural and Biological Sciences 14 12%
Neuroscience 8 7%
Engineering 4 3%
Medicine and Dentistry 4 3%
Other 6 5%
Unknown 32 27%
Attention Score in Context

Attention Score in Context

This research output has an Altmetric Attention Score of 32. 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 21 December 2022.
All research outputs
#1,261,285
of 25,576,275 outputs
Outputs from Genome Biology
#947
of 4,492 outputs
Outputs of similar age
#26,592
of 352,141 outputs
Outputs of similar age from Genome Biology
#25
of 80 outputs
Altmetric has tracked 25,576,275 research outputs across all sources so far. Compared to these this one has done particularly well and is in the 95th percentile: it's in the top 5% of all research outputs ever tracked by Altmetric.
So far Altmetric has tracked 4,492 research outputs from this source. They typically receive a lot more attention than average, with a mean Attention Score of 27.6. This one has done well, scoring higher than 78% 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 352,141 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 80 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 70% of its contemporaries.