Evidence map›Paper›PMID 31203387›Full record

ReviewMammalian genome : official journal of the International Mammalian Genome Society2019

Uses for humanised mouse models in precision medicine for neurodegenerative disease.

Remya R Nair, Silvia Corrochano, Samanta Gasco, Charlotte Tibbit, David Thompson, Cheryl Maduro, Zeinab Ali, Pietro Fratta, Abraham Acevedo Arozena, Thomas J Cunningham and 1 more

Open access · hybridAbstract readReview
In one paragraph

Review in Mammalian genome : official journal of the International Mammalian Genome Society, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 16 papers.

0numbers the graph read from it
0cells of the map it votes in
16citing papers in PubMed
2.0field-weighted citation impact, top 14% 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

16 citing papers in PubMed, 43 citations in OpenAlex.

  1. Review
  2. Preclinical research (on rare diseases): we need to talk about health equity.Mammalian genome : official journal of the International Mammalian Genome Society · 2025
    Review
  3. Article
  4. Review
  5. Review
  6. Review
  7. Article
  8. Tracking human neurologic disease status in mouse brain/plasma using reporter-tagged, EV-associated biomarkers.Molecular therapy : the journal of the American Society of Gene Therapy · 2023
    Article
  9. Review
  10. Article
  11. Article
  12. Article
  13. Article
  14. Review
  15. Article
  16. Neuromuscular disease modeling on a chip.Disease models & mechanisms · 2020
    Review
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

11 authors at 3 institutions in 2 countries.

Remya R NairMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
Silvia CorrochanoMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
Samanta GascoMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
Charlotte TibbitMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
David ThompsonMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
Cheryl MaduroDepartment of Neuromuscular Diseases, Institute of Neurology, University College London, London, WC1N 3BG, UK.
Zeinab AliMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK.
Pietro FrattaDepartment of Neuromuscular Diseases, Institute of Neurology, University College London, London, WC1N 3BG, UK.
Abraham Acevedo ArozenaUnidad de Investigación Hospital Universitario de Canarias, FUNCANIS, Instituto de Tecnologías Biomédicas ULL, and CIBERNED, La Laguna, 38320, Tenerife, Spain.
Thomas J CunninghamMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK. t.cunningham@har.mrc.ac.uk.ORCID 0000-0002-4751-3140
Elizabeth M C FisherMammalian Genetics Unit, MRC Harwell Institute, Oxfordshire, OX11 0RD, UK. elizabeth.fisher@ucl.ac.uk.ORCID 0000-0003-2850-9936
Mary Lyon Centre at MRC Harwell · GBUniversity College London · GBHospital Universitario de Canarias · ES

Funding

Medical Research Council MC_EX_MR/N501931/1Medical Research Council MR/L021056/1Medical Research Council MR/M008606/1Medical Research Council MR/S006508/1Motor Neurone Disease Association FISHER/APR14/874-792Motor Neurone Disease Association FISHER/OCT14/876-792Motor Neurone Disease Association FRATTA/JAN15/946-795Wellcome Trust 098327
6 · The paper itself

Abstract

Neurodegenerative disease encompasses a wide range of disorders afflicting the central and peripheral nervous systems and is a major unmet biomedical need of our time. There are very limited treatments, and no cures, for most of these diseases, including Alzheimer's Disease, Parkinson's Disease, Huntington Disease, and Motor Neuron Diseases. Mouse and other animal models provide hope by analysing them to understand pathogenic mechanisms, to identify drug targets, and to develop gene therapies and stem cell therapies. However, despite many decades of research, virtually no new treatments have reached the clinic. Increasingly, it is apparent that human heterogeneity within clinically defined neurodegenerative disorders, and between patients with the same genetic mutations, significantly impacts disease presentation and, potentially, therapeutic efficacy. Therefore, stratifying patients according to genetics, lifestyle, disease presentation, ethnicity, and other parameters may hold the key to bringing effective therapies from the bench to the clinic. Here, we discuss genetic and cellular humanised mouse models, and how they help in defining the genetic and environmental parameters associated with neurodegenerative disease, and so help in developing effective precision medicine strategies for future healthcare.

Indexed as

Disease Models, AnimalPrecision MedicineAnimalsChimeraHumansMiceMice, TransgenicNeurodegenerative DiseasesPhenotype

Identifiers

PMID31203387
PMCPMC6759662
OpenAlexW2950068635

What OpenQuestion holds

Textmetadata
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.