Evidence map›Paper›PMID 30250142›Full record

ArticleNature medicine2018

Genome editing in mitochondria corrects a pathogenic mtDNA mutation in vivo.

Payam A Gammage, Carlo Viscomi, Marie-Lune Simard, Ana S H Costa, Edoardo Gaude, Christopher A Powell, Lindsey Van Haute, Beverly J McCann, Pedro Rebelo-Guiomar, Raffaele Cerutti and 6 more

Open access · greenAbstract read
In one paragraph

Article in Nature medicine, 2018. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 180 papers.

0numbers the graph read from it
0cells of the map it votes in
180citing papers in PubMed
12.5field-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

180 citing papers in PubMed, 302 citations in OpenAlex.

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  20. Therapies for Mitochondrial Disease: Past, Present, and Future.Journal of inherited metabolic disease · 2025
    Review

120 more citing papers are in PubMed but not listed here.

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

16 authors at 4 institutions in 4 countries.

Payam A GammageMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK. payam.gammage@mrc-mbu.cam.ac.uk.ORCID http://orcid.org/0000-0003-1968-1726
Carlo ViscomiMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.ORCID http://orcid.org/0000-0001-6050-0566
Marie-Lune SimardMax Planck Institute for Biology of Ageing, Cologne, Germany.
Ana S H CostaMRC Cancer Unit, University of Cambridge, Cambridge, UK.
Edoardo GaudeMRC Cancer Unit, University of Cambridge, Cambridge, UK.
Christopher A PowellMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Lindsey Van HauteMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Beverly J McCannMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Pedro Rebelo-GuiomarMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Raffaele CeruttiMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Lei ZhangSangamo Therapeutics Inc., Richmond, CA, USA.
Edward J RebarSangamo Therapeutics Inc., Richmond, CA, USA.
Massimo ZevianiMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK.
Christian FrezzaMRC Cancer Unit, University of Cambridge, Cambridge, UK.ORCID http://orcid.org/0000-0002-3293-7397
James B StewartMax Planck Institute for Biology of Ageing, Cologne, Germany.ORCID http://orcid.org/0000-0002-2902-4968
Michal MinczukMRC Mitochondrial Biology Unit, University of Cambridge, Cambridge, UK. michal.minczuk@mrc-mbu.cam.ac.uk.ORCID http://orcid.org/0000-0001-8242-1420
University of Cambridge · GBMax Planck Institute for Biology of Ageing · DESangamo BioSciences (United States) · USUniversidade do Porto · PT

Funding

European Research Council 322424Medical Research Council MC_U105697135Medical Research Council MC_UP_1002/1Medical Research Council MC_UU_00015/4Medical Research Council MC_UU_00015/5Medical Research Council MC_UU_00015/8Medical Research Council MC_UU_12022/6
6 · The paper itself

Abstract

Mutations of the mitochondrial genome (mtDNA) underlie a substantial portion of mitochondrial disease burden. These disorders are currently incurable and effectively untreatable, with heterogeneous penetrance, presentation and prognosis. To address the lack of effective treatment for these disorders, we exploited a recently developed mouse model that recapitulates common molecular features of heteroplasmic mtDNA disease in cardiac tissue: the m.5024C>T tRNA

Indexed as

Gene EditingAnimalsDependovirusDisease Models, AnimalDNA, MitochondrialHumansMiceMitochondria, HeartMitochondrial DiseasesMutationPrognosisRNA, TransferZinc Finger NucleasesDNA, MitochondrialRNA, TransferZinc Finger Nucleases

Identifiers

PMID30250142
PMCPMC6225988
OpenAlexW2891993984

What OpenQuestion holds

Textmetadata
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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.