Evidence map›Paper›PMID 34703837›Full record

ArticleMolecular therapy. Methods & clinical development2021

Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.

Julien Baruteau, Sharon C Cunningham, Berna Seker Yilmaz, Dany P Perocheau, Simon Eaglestone, Derek Burke, Adrian J Thrasher, Simon N Waddington, Leszek Lisowski, Ian E Alexander and 1 more

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

Article in Molecular therapy. Methods & clinical development, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 18 papers.

0numbers the graph read from it
0cells of the map it votes in
18citing papers in PubMed
4.7field-weighted citation impact, top 5% 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

18 citing papers in PubMed, 31 citations in OpenAlex.

  1. Review
  2. Article
  3. The deLIVERed promises of gene therapy: Past, present, and future of liver-directed gene therapy.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Review
  4. Gene Therapy for Inherited Liver Disease: To Add or to Edit.International journal of molecular sciences · 2024
    Review
  5. Article
  6. Review
  7. Review
  8. Article
  9. Article
  10. Review
  11. Gene therapy for liver diseases - progress and challenges.Nature reviews. Gastroenterology & hepatology · 2023
    Review
  12. Article
  13. Article
  14. Review
  15. Review
  16. Article
  17. Article
  18. 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

11 authors at 5 institutions in 5 countries.

Julien BaruteauGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
Sharon C CunninghamGene Therapy Research Unit, Children's Medical Research Institute and Children's Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney, Sydney, Australia.
Berna Seker YilmazGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
Dany P PerocheauGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
Simon EaglestoneTranslational Research Office, University College London, London, UK.
Derek BurkeEnzyme Unit, NIHR BRC, Great Ormond Street Hospital Foundation Trust and UCL Great Ormond Street Institute of Child Health, London, UK.
Adrian J ThrasherMolecular & Cellular Immunology, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
Simon N WaddingtonGene Transfer Technology Group, Institute for Women's Health, University College London, 86-96 Chenies Mews, London, UK.
Leszek LisowskiTranslational Vectorology Unit, Children's Medical Research Institute, The University of Sydney, Westmead, NSW, Australia.
Ian E AlexanderGene Therapy Research Unit, Children's Medical Research Institute and Children's Hospital at Westmead, Faculty of Medicine and Health, The University of Sydney, Sydney, Australia.
Paul GissenGenetics and Genomic Medicine Department, Great Ormond Street Institute of Child Health, University College London, London WC1N 1EH, UK.
Great Ormond Street Hospital · GBThe University of Sydney · AUGreat Ormond Street Hospital for Children NHS Foundation Trust · GBUniversity College London · GBUniversity of the Witwatersrand · ZA

Funding

Medical Research Council MR/T008024/1
6 · The paper itself

Abstract

X-linked inherited ornithine transcarbamylase deficiency (OTCD) is the most common disorder affecting the liver-based urea cycle, a pathway enabling detoxification of nitrogen waste and endogenous arginine biosynthesis. Patients develop acute hyperammonemia leading to neurological sequelae or death despite the best-accepted therapy based on ammonia scavengers and protein-restricted diet. Liver transplantation is curative but associated with procedure-related complications and lifelong immunosuppression. Adeno-associated viral (AAV) vectors have demonstrated safety and clinical benefits in a rapidly growing number of clinical trials for inherited metabolic liver diseases. Engineered AAV capsids have shown promising enhanced liver tropism. Here, we conducted a good-laboratory practice-compliant investigational new drug-enabling study to assess the safety of intravenous liver-tropic AAVLK03 gene transfer of a human codon-optimized

Indexed as

AAVAAVLK03adeno-associated virusbiodistributioncynomolgus macaqueengineered capsidimmune responselivernon-human primatesornithine transcarbamylase deficiency

Identifiers

PMID34703837
PMCPMC8517016
OpenAlexW3200805144

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.