Evidence map›Paper›PMID 33369168›Full record

ArticleEMBO molecular medicine2021

Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders.

Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta and 9 more

Open access · goldAbstract read
In one paragraph

Article in EMBO molecular medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 15 papers.

0numbers the graph read from it
0cells of the map it votes in
15citing papers in PubMed
2.3field-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

15 citing papers in PubMed, 28 citations in OpenAlex.

  1. Science advances · 2026
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  7. Modelling urea cycle disorders using iPSCs.NPJ Regenerative medicine · 2022
    Review
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  13. Autophagy in major human diseases.The EMBO journal · 2021
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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

19 authors at 6 institutions in 3 countries.

Leandro R SoriaTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.ORCID 0000-0002-7124-856X
Sonam GurungUCL Great Ormond Street Institute of Child Health, London, UK.
Giulia De SabbataInternational Centre for Genetic Engineering and Biotechnology, Trieste, Italy.
Dany P PerocheauUCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0001-5450-8801
Angela De AngelisTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.
Gemma BrunoTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.
Elena PolishchukTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.
Debora ParisInstitute of Biomolecular Chemistry, National Research Council, Pozzuoli, Italy.
Paola CuomoInstitute of Biomolecular Chemistry, National Research Council, Pozzuoli, Italy.
Andrea MottaInstitute of Biomolecular Chemistry, National Research Council, Pozzuoli, Italy.
Michael OrfordUCL Great Ormond Street Institute of Child Health, London, UK.
Youssef KhalilUCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0001-9025-3017
Simon EatonUCL Great Ormond Street Institute of Child Health, London, UK.
Philippa B MillsUCL Great Ormond Street Institute of Child Health, London, UK.
Simon N WaddingtonUCL Great Ormond Street Institute of Child Health, London, UK.
Carmine SettembreTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.ORCID 0000-0002-5829-8589
Andrés F MuroInternational Centre for Genetic Engineering and Biotechnology, Trieste, Italy.ORCID 0000-0002-9628-0494
Julien BaruteauUCL Great Ormond Street Institute of Child Health, London, UK.ORCID 0000-0003-0582-540X
Nicola Brunetti-PierriTelethon Institute of Genetics and Medicine, Pozzuoli, Italy.ORCID 0000-0002-6895-8819
Great Ormond Street Hospital · GBTelethon Institute Of Genetics And Medicine · ITNational Research Council · ITInternational Centre for Genetic Engineering and Biotechnology · ITFederico II University Hospital · ITSouth African Medical Research Council · ZA

Funding

Department of HealthMedical Research Council MR/T008024/1
6 · The paper itself

Abstract

Urea cycle disorders (UCD) are inherited defects in clearance of waste nitrogen with high morbidity and mortality. Novel and more effective therapies for UCD are needed. Studies in mice with constitutive activation of autophagy unravelled Beclin-1 as druggable candidate for therapy of hyperammonemia. Next, we investigated efficacy of cell-penetrating autophagy-inducing Tat-Beclin-1 (TB-1) peptide for therapy of the two most common UCD, namely ornithine transcarbamylase (OTC) and argininosuccinate lyase (ASL) deficiencies. TB-1 reduced urinary orotic acid and improved survival under protein-rich diet in spf-ash mice, a model of OTC deficiency (proximal UCD). In Asl

Indexed as

Argininosuccinic AciduriaOrnithine Carbamoyltransferase Deficiency DiseaseUrea Cycle Disorders, InbornAnimalsAutophagyBeclin-1MiceBeclin-1argininosuccinic aciduriaautophagyOTC deficiencyTat-Beclin-1 peptideurea cycle disorders

Identifiers

PMID33369168
PMCPMC7863400
OpenAlexW3113672812

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.