Evidence map›Paper›PMID 39844454›Full record

ArticleNucleic acids research2025

Tissue-specific modulation of CRISPR activity by miRNA-sensing guide RNAs.

Antonio Garcia-Guerra, Chaitra Sathyaprakash, Olivier G de Jong, Wooi F Lim, Pieter Vader, Samir El Andaloussi, Jonathan Bath, Jesus Reine, Yoshitsugu Aoki, Andrew J Turberfield and 2 more

Abstract read
In one paragraph

Article in Nucleic acids research, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 7 papers.

0numbers the graph read from it
0cells of the map it votes in
7citing papers in PubMed
–field-weighted citation impact
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

7 citing papers in PubMed.

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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

12 authors.

Antonio Garcia-GuerraDepartment of Physics, University of Oxford, OX1 3PU Oxford, United Kingdom.
Chaitra SathyaprakashDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, 187-8551 Tokyo, Japan.
Olivier G de JongDepartment of Pharmaceutics, Utrecht Institute of Pharmaceutical Sciences, Utrecht University, 3584 CG Utrecht, The Netherlands.
Wooi F LimDepartment of Paediatrics, University of Oxford, OX3 7TY Oxford, United Kingdom.
Pieter VaderCDL Research, University Medical Center Utrecht, 3584 CX Utrecht, The Netherlands.ORCID 0000-0002-7059-8920
Samir El AndaloussiDepartment of Laboratory Medicine, TRACK, Karolinska Institutet, 141 52 Huddinge, Sweden.
Jonathan BathDepartment of Physics, University of Oxford, OX1 3PU Oxford, United Kingdom.
Jesus ReineOxford Vaccine Group, University of Oxford, OX3 7LE Oxford, United Kingdom.
Yoshitsugu AokiDepartment of Molecular Therapy, National Institute of Neuroscience, National Centre of Neurology and Psychiatry, 187-8551 Tokyo, Japan.
Andrew J TurberfieldDepartment of Physics, University of Oxford, OX1 3PU Oxford, United Kingdom.
Matthew J A WoodDepartment of Paediatrics, University of Oxford, OX3 7TY Oxford, United Kingdom.
Carlo RinaldiDepartment of Paediatrics, University of Oxford, OX3 7TY Oxford, United Kingdom.ORCID 0000-0002-8307-6638

Funding

Kennedy's Disease AssociationLa Caixa Foundation Fellowship LCF/BQ/EU16/11560044Marie Curie Initial Training Network EScoDNA 317110Medical Research Council MR/N024850/1Medical Research Council MR/Y009703/1Muscular Dystrophy Association 578222University of Oxfordxford University Press John Fell Fund
6 · The paper itself

Abstract

Nucleic acid nanostructures offer unique opportunities for biomedical applications due to their sequence-programmable structures and functions, which enable the design of complex responses to molecular cues. Control of the biological activity of therapeutic cargoes based on endogenous molecular signatures holds the potential to overcome major hurdles in translational research: cell specificity and off-target effects. Endogenous microRNAs (miRNAs) can be used to profile cell type and cell state, and are ideal inputs for RNA nanodevices. Here, we present CRISPR MiRAGE (miRNA-activated genome editing), a tool comprising a dynamic single-guide RNA that senses miRNA complexed with Argonaute proteins and controls downstream CRISPR (Clustered Regularly Interspaced Short Palindromic Repeats) activity based on the detected miRNA signature. We study the operation of the miRNA-sensing single-guide RNA and attain muscle-specific activation of gene editing through CRISPR MiRAGE in models of Duchenne muscular dystrophy. By enabling RNA-controlled gene editing activity, this technology creates opportunities to advance tissue-specific CRISPR treatments for human diseases.

Indexed as

Clustered Regularly Interspaced Short Palindromic RepeatsCRISPR-Cas SystemsGene EditingMicroRNAsRNA, Guide, CRISPR-Cas SystemsAnimalsArgonaute ProteinsHEK293 CellsHumansMiceMuscular Dystrophy, DuchenneOrgan SpecificityArgonaute ProteinsMicroRNAsRNA, Guide, CRISPR-Cas Systems

Identifiers

PMID39844454
PMCPMC11754125

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Registered trials

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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.