Evidence map›Paper›PMID 29551272›Full record

ArticleCell2018

Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors.

Silvana Konermann, Peter Lotfy, Nicholas J Brideau, Jennifer Oki, Maxim N Shokhirev, Patrick D Hsu

Open access · bronzeAbstract read
In one paragraph

Article in Cell, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 729 papers, 1 of them a synthesis that pooled it.

0numbers the graph read from it
0cells of the map it votes in
729citing papers in PubMed, 1 pooled it
43.3field-weighted citation impact, top 1% 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

729 citing papers in PubMed, 1 synthesis or guideline pooled it, 1,188 citations in OpenAlex.

  1. Pooled it
  2. Article
  3. Review
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  6. Article
  7. Cap-Independent Translation Regulation of VEGF Using CRISPR-dCas13d.bioRxiv : the preprint server for biology · 2026
    Article
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669 more citing papers are in PubMed but not listed here.

4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

6 authors at 1 institution in 1 country.

Silvana KonermannLaboratory of Molecular and Cell Biology, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA; Helmsley Center for Genomic Medicine, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA.
Peter LotfyLaboratory of Molecular and Cell Biology, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA; Helmsley Center for Genomic Medicine, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA.
Nicholas J BrideauLaboratory of Molecular and Cell Biology, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA; Helmsley Center for Genomic Medicine, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA.
Jennifer OkiLaboratory of Molecular and Cell Biology, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA; Helmsley Center for Genomic Medicine, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA.
Maxim N ShokhirevRazavi Newman Integrative Genomics and Bioinformatics Core, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA.
Patrick D HsuLaboratory of Molecular and Cell Biology, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA; Helmsley Center for Genomic Medicine, Salk Institute for Biological Studies, 10010 N. Torrey Pines Road, La Jolla, CA 92037, USA. Electronic address: patrick@salk.edu.
Salk Institute for Biological Studies · US

Funding

Viral Vector Core (VVC)P30CA014195 · NCI · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI Ye Zheng · 1985 to 2026
$82.8M
Salk Institute for Biological Studies NINDS Center Core GrantP30NS072031 · NINDS · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI O'LEARY, DENNIS D · 2011 to 2015
$4.2M
Eukaryotic transcriptome engineering via sequence-specific regulation of endogenous RNADP5OD021369 · OD · UNIVERSITY OF CALIFORNIA BERKELEY · PI HSU, PATRICK · 2015 to 2018
$2.4M
Molecular tools for targeting RNAR01GM131073 · NIGMS · UNIVERSITY OF CALIFORNIA BERKELEY · PI HSU, PATRICK · 2019 to 2022
$1.3M
Mechanistic studies of RNA-targeting CRISPR systemsR01GM132465 · NIGMS · UNIVERSITY OF CALIFORNIA BERKELEY · PI HSU, PATRICK, LYUMKIS, DMITRY · 2020 to 2022
$908k
Genome-scale genetic perturbation of the APOE2 and APOE4 human interactomesR21AG056811 · NIA · SALK INSTITUTE FOR BIOLOGICAL STUDIES · PI HSU, PATRICK · 2017 to 2018
$534k
NCI NIH HHS P30 CA014195NIA NIH HHS R21 AG056811NIGMS NIH HHS R01 GM131073NIGMS NIH HHS R01 GM132465NIH HHS DP5 OD021369NINDS NIH HHS P30 NS072031
6 · The paper itself

Abstract

Class 2 CRISPR-Cas systems endow microbes with diverse mechanisms for adaptive immunity. Here, we analyzed prokaryotic genome and metagenome sequences to identify an uncharacterized family of RNA-guided, RNA-targeting CRISPR systems that we classify as type VI-D. Biochemical characterization and protein engineering of seven distinct orthologs generated a ribonuclease effector derived from Ruminococcus flavefaciens XPD3002 (CasRx) with robust activity in human cells. CasRx-mediated knockdown exhibits high efficiency and specificity relative to RNA interference across diverse endogenous transcripts. As one of the most compact single-effector Cas enzymes, CasRx can also be flexibly packaged into adeno-associated virus. We target virally encoded, catalytically inactive CasRx to cis elements of pre-mRNA to manipulate alternative splicing, alleviating dysregulated tau isoform ratios in a neuronal model of frontotemporal dementia. Our results present CasRx as a programmable RNA-binding module for efficient targeting of cellular RNA, enabling a general platform for transcriptome engineering and future therapeutic development.

Indexed as

CRISPR-Cas SystemsAlternative SplicingAnimalsBacterial ProteinsCell DifferentiationComputational BiologyEscherichia coliGene Expression ProfilingGenetic EngineeringHEK293 CellsHumansInduced Pluripotent Stem CellsLentivirusMiceProtein EngineeringRNABacterial ProteinsRNARNA, Guide, CRISPR-Cas Systemsalternative splicingCas13CasRxCRISPRfrontotemporal dementiagene editinggenome engineeringRNA interferenceRNA targetingtau

Identifiers

PMID29551272
PMCPMC5910255
OpenAlexW2793359586

What OpenQuestion holds

Textmetadata
LicenceTDM
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.