Evidence map›Paper›PMID 27190030›Full record

ReviewBrain : a journal of neurology2016

Emerging therapies for mitochondrial disorders.

Helen Nightingale, Gerald Pfeffer, David Bargiela, Rita Horvath, Patrick F Chinnery

Abstract readReview
In one paragraph

Review in Brain : a journal of neurology, 2016. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 26 papers.

0numbers the graph read from it
0cells of the map it votes in
26citing 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

26 citing papers in PubMed.

  1. Review
  2. Article
  3. Precision mitochondrial medicine.Cambridge prisms. Precision medicine · 2023
    Review
  4. Review
  5. Review
  6. Chronic pain is common in mitochondrial disease.Neuromuscular disorders : NMD · 2020
    Article
  7. Observational
  8. Article
  9. Article
  10. Advances in primary mitochondrial myopathies.Current opinion in neurology · 2019
    Review
  11. Article
  12. Article
  13. Review
  14. Article
  15. Article
  16. The genetic basis of disease.Essays in biochemistry · 2018
    Review
  17. Review
  18. Article
  19. Article
  20. Article
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

5 authors.

Helen NightingaleWellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Newcastle University, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK.
Gerald PfefferWellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Newcastle University, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK Department of Clinical Neurosciences, University of Calgary, Calgary, Canada Hotchkiss Brain Institute, at the University of Calgary, Calgary, Canada.
David BargielaWellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Newcastle University, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK.
Rita HorvathWellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Newcastle University, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK.
Patrick F ChinneryWellcome Trust Centre for Mitochondrial Research, Institute of Genetic Medicine, Newcastle University, Central Parkway, Newcastle upon Tyne, NE1 3BZ, UK MRC-Mitochondrial Biology Unit, Cambridge Biomedical Campus, Cambridge, CB2 0XY, UK Department of Clinical Neurosciences, Cambridge Biomedical Campus, University of Cambridge, Cambridge, CB2 0QQ, UK pfc25@medschl.cam.ac.uk.

Funding

Medical Research Council G0601943Medical Research Council G1000848Medical Research Council MC_PC_13047Medical Research Council MC_UP_1501/2Medical Research Council MR/N025431/1Wellcome TrustWellcome Trust 096919Z/11/ZWellcome Trust 101876/Z/13/Z
6 · The paper itself

Abstract

Mitochondrial disorders are a diverse group of debilitating conditions resulting from nuclear and mitochondrial DNA mutations that affect multiple organs, often including the central and peripheral nervous system. Despite major advances in our understanding of the molecular mechanisms, effective treatments have not been forthcoming. For over five decades patients have been treated with different vitamins, co-factors and nutritional supplements, but with no proven benefit. There is therefore a clear need for a new approach. Several new strategies have been proposed acting at the molecular or cellular level. Whilst many show promise in vitro, the clinical potential of some is questionable. Here we critically appraise the most promising preclinical developments, placing the greatest emphasis on diseases caused by mitochondrial DNA mutations. With new animal and cellular models, longitudinal deep phenotyping in large patient cohorts, and growing interest from the pharmaceutical industry, the field is poised to make a breakthrough.

Indexed as

AnimalsDisease Models, AnimalDNA, MitochondrialDrug Delivery SystemsHumansMitochondrial DiseasesModels, BiologicalMolecular Targeted TherapyMutationStem Cell TransplantationDNA, Mitochondrialgene therapiesmitochondrial disorderspharmaceuticalsproteintreatment

Identifiers

PMID27190030
PMCPMC4892756

What OpenQuestion holds

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LicenceCC BY
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.