Evidence map›Paper›PMID 38618017›Full record

ArticleF1000Research2023

Dany Perocheau, Sonam Gurung, Loukia Touramanidou, Claire Duff, Garima Sharma, Neil Sebire, Patrick F Finn, Alex Cavedon, Summar Siddiqui, Lisa Rice and 3 more

Open access · goldAbstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in F1000Research, 2023. 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
2.3field-weighted citation impact, top 13% 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

7 citing papers in PubMed, 6 citations in OpenAlex.

  1. New approach methodologies (NAMs) for preclinical and translational evaluation of mRNA-lipid nanoparticle (LNP) therapeutics.Journal of controlled release : official journal of the Controlled Release Society · 2026
    Review
  2. Article
  3. Review
  4. Article
  5. Exploring RNA therapeutics for urea cycle disorders.Journal of inherited metabolic disease · 2024
    Review
  6. Review
  7. F1000Research · 2023
    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

13 authors at 3 institutions in 3 countries.

Dany PerocheauGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.ORCID https://orcid.org/0000-0001-5450-8801
Sonam GurungGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.
Loukia TouramanidouGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.
Claire DuffGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.
Garima SharmaGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.
Neil SebireGreat Ormond Street Hospital for Children NHS Foundation Trust, London, England, WC1N 3JH, UK.
Patrick F FinnModerna Inc., Cambridge, MA 02139, USA.ORCID https://orcid.org/0000-0003-1333-5400
Alex CavedonModerna Inc., Cambridge, MA 02139, USA.
Summar SiddiquiModerna Inc., Cambridge, MA 02139, USA.
Lisa RiceModerna Inc., Cambridge, MA 02139, USA.
Paolo G V MartiniModerna Inc., Cambridge, MA 02139, USA.
Andrea FrassettoModerna Inc., Cambridge, MA 02139, USA.
Julien BaruteauGreat Ormond Street Institute of Child Health, University College London, London, England, WC1N 1EH, UK.
Great Ormond Street Hospital · GBModerna Therapeutics (United States) · USGreat Ormond Street Hospital for Children NHS Foundation Trust · GB

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

Background: In academic research and the pharmaceutical industry, Methods: Here, we describe an optimised and easy-to-implement protocol for the culture of sections from mouse livers, enabling its use as a reliable Results: We show that precision-cut liver sections can be a reliable model for recapitulating the biological phenotype of inherited metabolic diseases, exemplified by common urea cycle defects such as citrullinemia type 1 and argininosuccinic aciduria, caused by argininosuccinic synthase (ASS1) and argininosuccinic lyase (ASL) deficiencies respectively. Conclusions: Therapeutic response to gene therapy such as messenger RNA replacement delivered via lipid nanoparticles can be monitored, demonstrating that precision-cut liver sections can be used as a preclinical screening tool to assess therapeutic response and toxicity in monogenic liver diseases.

Indexed as

Liver DiseasesMetabolic DiseasesAnimalsMicePhenotypeReproducibility of ResultsArgininosuccinic aciduriaCitrullinemia type 1lipid nanoparticlelivermRNAprecision-cut tissue slicesurea cyclevibratome

Identifiers

PMID38618017
PMCPMC11016166
OpenAlexW4394576633

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.