Evidence map›Paper›PMID 38016974›Full record

ArticleScientific reports2023

Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.

Tjessa Bondue, Sante Princiero Berlingerio, Florian Siegerist, Elena Sendino-Garví, Maximilian Schindler, Hans Jacobus Baelde, Sara Cairoli, Bianca Maria Goffredo, Fanny Oliveira Arcolino, Jürgen Dieker and 6 more

Open access · goldAbstract read
In one paragraph

Article in Scientific reports, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 6 papers.

0numbers the graph read from it
0cells of the map it votes in
6citing papers in PubMed
2.6field-weighted citation impact, top 10% 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

6 citing papers in PubMed, 10 citations in OpenAlex.

  1. Review
  2. Article
  3. Long-term outcomes in nephropathic cystinosis: a review.Pediatric nephrology (Berlin, Germany) · 2026
    Review
  4. Article
  5. Review
  6. 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

16 authors at 8 institutions in 5 countries.

Tjessa BondueLaboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
Sante Princiero Berlingerio *Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
Florian Siegerist *Institute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
Elena Sendino-GarvíDivision Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Maximilian SchindlerInstitute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
Hans Jacobus BaeldeDepartment of Pathology, Leiden University Medical Center, Leiden, The Netherlands.ORCID 0000-0002-1214-500X
Sara CairoliLaboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Bianca Maria GoffredoLaboratory of Metabolic Biochemistry, Department of Pediatric Medicine, Bambino Gesù Children's Hospital, IRCCS, Rome, Italy.
Fanny Oliveira ArcolinoLaboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
Jürgen DiekerMercurna B.V., Oss, The Netherlands.
Manoe Jacoba JanssenDivision Pharmacology, Utrecht Institute for Pharmaceutical Sciences, Utrecht University, Utrecht, The Netherlands.
Nicole EndlichInstitute of Anatomy and Cell Biology, Universitätsmedizin Greifswald, Greifswald, Germany.
Roland BrockDepartment of Medical Biosciences, Radboud University Medical Center, Nijmegen, The Netherlands.
Rik GijsbersLaboratory for Molecular Virology and Gene Therapy, Department of Pharmaceutical and Pharmacological Sciences, KU Leuven, Leuven, Belgium.
Lambertus van den Heuvel *Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.
Elena Levtchenko *Laboratory of Pediatric Nephrology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium. e.n.levtchenko@amsterdamumc.nl.
KU Leuven · BEUniversitätsmedizin Greifswald · DEBambino Gesù Children's Hospital · ITEmma Kinderziekenhuis · NLRadboud University Nijmegen · NLUtrecht University · NLLeiden University Medical Center · NLMerck (Netherlands) · NL

Funding

Bundesministerium für Bildung und Forschung (Federal Ministry of Education and Research) 01GM1518BBundesministerium für Bildung und Forschung (Federal Ministry of Education and Research) STOP-FSGSFonds Wetenschappelijk Onderzoek (Research Foundation Flanders) 11A7821NFonds Wetenschappelijk Onderzoek (Research Foundation Flanders) 11A7823NFonds Wetenschappelijk Onderzoek (Research Foundation Flanders) 1801120NKU Leuven (Katholieke Universiteit Leuven) C14/17/11
6 · The paper itself

Abstract

Messenger RNA (mRNA) therapies are emerging in different disease areas, but have not yet reached the kidney field. Our aim was to study the feasibility to treat the genetic defect in cystinosis using synthetic mRNA in cell models and ctns

Indexed as

Amino Acid Transport Systems, NeutralCystinosisAnimalsCystineDietary SupplementsModels, TheoreticalRNA, MessengerZebrafishAmino Acid Transport Systems, NeutralCystineRNA, Messenger

Identifiers

PMID38016974
PMCPMC10684520
OpenAlexW4389085087

What Socratic 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 Socratic graph.