Evidence map›Paper›PMID 31439808›Full record

ArticleRNA (New York, N.Y.)2019

Tissue-restricted genome editing in vivo specified by microRNA-repressible anti-CRISPR proteins.

Jooyoung Lee, Haiwei Mou, Raed Ibraheim, Shun-Qing Liang, Pengpeng Liu, Wen Xue, Erik J Sontheimer

Open access · bronzeAbstract read
In one paragraph

Article in RNA (New York, N.Y.), 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 59 papers.

0numbers the graph read from it
0cells of the map it votes in
59citing papers in PubMed
4.2field-weighted citation impact, top 5% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.

3 · Its place in the literature

Who cites it

59 citing papers in PubMed, 98 citations in OpenAlex.

  1. Review
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  8. Review
  9. Protective antigen-mediated delivery of an anti-CRISPR protein for precision genome editing.Proceedings of the National Academy of Sciences of the United States of America · 2025
    Article
  10. Article
  11. Article
  12. Review
  13. Mechanism of Cas9 inhibition by AcrIIA11.Nucleic acids research · 2025
    Article
  14. Article
  15. Article
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  17. Comprehensive deletion scan of anti-CRISPR AcrIIA4 reveals essential and dispensable domains for Cas9 inhibition.Proceedings of the National Academy of Sciences of the United States of America · 2024
    Article
  18. Article
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4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

7 authors at 1 institution in 1 country.

Jooyoung LeeRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0003-4752-3792
Haiwei MouRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0002-1710-1882
Raed IbraheimRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0002-0961-7299
Shun-Qing LiangRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0003-4664-6029
Pengpeng LiuProgram in Molecular, Cell and Cancer Biology, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0002-4891-8780
Wen XueRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0002-9797-8042
Erik J SontheimerRNA Therapeutics Institute, University of Massachusetts Medical School, Worcester, Massachusetts 01605, USA.ORCID 0000-0002-0881-0310
University of Massachusetts Chan Medical School · US

Funding

Viral Vector CoreP01HL131471 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI XUE, WEN · 2016 to 2020
$11.3M
Develop combinatorial non-viral and viral CRISPR delivery for lung diseasesUG3HL147367 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI ANDERSON, DANIEL G, GAO, GUANGPING · 2018 to 2020
$2.7M
CRISPR-based modular therapy for precision medicineDP2HL137167 · NHLBI · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI XUE, WEN · 2016 to 2016
$2.5M
Enhancing Genome Editing Technology with Natural Cas9 InhibitorsR01GM125797 · NIGMS · UNIV OF MASSACHUSETTS MED SCH WORCESTER · PI SONTHEIMER, ERIK J. · 2018 to 2021
$1.4M
NHLBI NIH HHS DP2 HL137167NHLBI NIH HHS P01 HL131471NHLBI NIH HHS UG3 HL147367NIGMS NIH HHS R01 GM125797
6 · The paper itself

Abstract

CRISPR-Cas systems are bacterial adaptive immune pathways that have revolutionized biotechnology and biomedical applications. Despite the potential for human therapeutic development, there are many hurdles that must be overcome before its use in clinical settings. Some clinical safety concerns arise from editing activity in unintended cell types or tissues upon in vivo delivery (e.g., by adeno-associated virus (AAV) vectors). Although tissue-specific promoters and serotypes with tissue tropisms can be used, suitably compact promoters are not always available for desired cell types, and AAV tissue tropism specificities are not absolute. To reinforce tissue-specific editing, we exploited anti-CRISPR proteins (Acrs) that have evolved as natural countermeasures against CRISPR immunity. To inhibit Cas9 in all ancillary tissues without compromising editing in the target tissue, we established a flexible platform in which an

Indexed as

CRISPR-Cas SystemsGene EditingAnimalsDependovirusGenetic VectorsHEK293 CellsHumansMiceMicroRNAsPromoter Regions, GeneticRNA, Guide, CRISPR-Cas SystemsTransgenesMicroRNAsRNA, Guide, CRISPR-Cas SystemsAAVanti-CRISPRCas9microRNAtissue-specific editing

Identifiers

PMID31439808
PMCPMC6795140
OpenAlexW2969305945

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC
Read underepoch 390

Registered trials

None linked

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.