Evidence map›Paper›PMID 34108249›Full record

ArticleScience translational medicine2021

Therapeutic liver repopulation by transient acetaminophen selection of gene-modified hepatocytes.

Anne Vonada, Amita Tiyaboonchai, Sean Nygaard, Jeffrey Posey, Alexander Mack Peters, Shelley R Winn, Alessio Cantore, Luigi Naldini, Cary O Harding, Markus Grompe

Abstract read
In one paragraph

Article in Science translational medicine, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 19 papers.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed
–field-weighted citation impact
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

19 citing papers in PubMed.

  1. Review
  2. Minicells derived fromApplied and environmental microbiology · 2026
    Article
  3. Article
  4. Article
  5. Article
  6. Article
  7. Current state of the treatment landscape of phenylketonuria.Orphanet journal of rare diseases · 2025
    Review
  8. Article
  9. Repair Drive improves gene editing in the liver.Molecular therapy : the journal of the American Society of Gene Therapy · 2025
    Article
  10. Therapeutic liver cell transplantation to treat murine PKU.Journal of inherited metabolic disease · 2024
    Article
  11. Article
  12. Targeted Gene Insertion: The Cutting Edge of CRISPR Drug Development with Hemophilia as a Highlight.BioDrugs : clinical immunotherapeutics, biopharmaceuticals and gene therapy · 2024
    Review
  13. State-of-the-art 2023 on gene therapy for phenylketonuria.Journal of inherited metabolic disease · 2024
    Review
  14. Article
  15. Article
  16. Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice.Molecular therapy. Methods & clinical development · 2022
    Article
  17. Article
  18. Article
  19. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors.

Anne VonadaOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0003-0398-1898
Amita TiyaboonchaiOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0002-0116-7393
Sean NygaardOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0002-8239-6584
Jeffrey PoseyOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0001-5205-8261
Alexander Mack PetersOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA.
Shelley R WinnDepartment of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0002-3035-0584
Alessio CantoreSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.ORCID 0000-0002-9741-997X
Luigi NaldiniSan Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute, 20132 Milan, Italy.ORCID 0000-0002-7835-527X
Cary O HardingDepartment of Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR 97239, USA.ORCID 0000-0001-9146-3112
Markus GrompeOregon Stem Cell Center, Oregon Health & Science University, Portland, OR 97239, USA. grompem@ohsu.edu.ORCID 0000-0002-6616-4345

Funding

Treatment of Distal Tyrosine Pathway DisordersR01DK048252 · NIDDK · OREGON HEALTH & SCIENCE UNIVERSITY · PI GROMPE, MARKUS · 1994 to 2017
$5.5M
Program in Molecular and Cellular BiosciencesT32GM071338 · NIGMS · OREGON HEALTH & SCIENCE UNIVERSITY · PI MASLEN, CHERYL L · 2005 to 2020
$3.5M
In vivo selection of gene modified hepatocytesR01DK123093 · NIDDK · OREGON HEALTH & SCIENCE UNIVERSITY · PI GROMPE, MARKUS · 2021 to 2023
$1.5M
NIDDK NIH HHS R01 DK048252NIDDK NIH HHS R01 DK123093NIGMS NIH HHS T32 GM071338
6 · The paper itself

Abstract

Gene therapy by integrating vectors is promising for monogenic liver diseases, especially in children where episomal vectors remain transient. However, reaching the therapeutic threshold with genome-integrating vectors is challenging. Therefore, we developed a method to expand hepatocytes bearing therapeutic transgenes. The common fever medicine acetaminophen becomes hepatotoxic via cytochrome p450 metabolism. Lentiviral vectors with transgenes linked in cis to a

Indexed as

AcetaminophenHepatocytesAnimalsGenetic TherapyLiverMiceTransgenesAcetaminophen

Identifiers

PMID34108249
PMCPMC9094690

What OpenQuestion holds

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Registered trials

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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.