Evidence map›Paper›PMID 29724658›Full record

ArticleMolecular genetics and metabolism2018

Human hepatocyte transplantation corrects the inherited metabolic liver disorder arginase deficiency in mice.

Stephanie A K Angarita, Brian Truong, Suhail Khoja, Matthew Nitzahn, Abha K Rajbhandari, Irina Zhuravka, Sergio Duarte, Michael G Lin, Alex K Lam, Stephen D Cederbaum and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism, 2018. 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
0.9field-weighted citation impact, top 28% 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, 8 citations in OpenAlex.

  1. Article
  2. Emerging technologies and ethics-exogenic chimeric humanized organs.American journal of transplantation : official journal of the American Society of Transplantation and the American Society of Transplant Surgeons · 2022
    Article
  3. Review
  4. Current status of surviving patients with arginase 1 deficiency in Japan.Molecular genetics and metabolism reports · 2021
    Article
  5. Article
  6. Article
  7. Lipid nanoparticle-targeted mRNA therapy as a treatment for the inherited metabolic liver disorder arginase deficiency.Proceedings of the National Academy of Sciences of the United States of America · 2019
    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 1 institution in 1 country.

Stephanie A K AngaritaDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Brian TruongDepartment of Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Suhail KhojaDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Matthew NitzahnDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Molecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Abha K RajbhandariBehavioral Testing Core Facility, Department of Psychology and Brain Research Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Irina ZhuravkaBehavioral Testing Core Facility, Department of Psychology and Brain Research Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Sergio DuarteDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Michael G LinDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Alex K LamDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Stephen D CederbaumDepartment of Psychiatry, The David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Intellectual and Developmental Disabilities Research Center at UCLA, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States.
Gerald S LipshutzDepartment of Surgery, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Department of Molecular and Medical Pharmacology, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Molecular Biology Institute, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Department of Psychiatry, The David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Intellectual and Developmental Disabilities Research Center at UCLA, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Department of Medicine, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Department of Urology, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Semel Institute for Neuroscience, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States; Broad Center for Regenerative Medicine and Stem Cell Research at UCLA, David Geffen School of Medicine at UCLA, Los Angeles, CA, United States. Electronic address: glipshutz@mednet.ucla.edu.
University of California, Los Angeles · US

Funding

UCLA Medical Genetics Training ProgramT32GM008243 · NIGMS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI Julian Martinez-Agosto, STANLEY F. NELSON · 1987 to 2026
$6.8M
UCLA Center for Translational Research in Neurodevelopment: UC-TRaNU54HD087101 · NICHD · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI MCCRACKEN, JAMES T. · 2016 to 2020
$6.5M
Interdisciplinary Training in Virology and Gene TherapyT32AI060567 · NIAID · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI WU, TING-TING · 2004 to 2018
$3.4M
Cell and Gene Replacement Strategies for Arginase DeficiencyR01NS100979 · NINDS · UNIVERSITY OF CALIFORNIA LOS ANGELES · PI LIPSHUTZ, GERALD S · 2017 to 2021
$1.7M
NIAID NIH HHS T32 AI060567NICHD NIH HHS U54 HD087101NINDS NIH HHS R01 NS100979
6 · The paper itself

Abstract

The transplantation, engraftment, and expansion of primary hepatocytes have the potential to be an effective therapy for metabolic disorders of the liver including those of nitrogen metabolism. To date, such methods for the treatment of urea cycle disorders in murine models has only been minimally explored. Arginase deficiency, an inherited disorder of nitrogen metabolism that presents in the first two years of life, has the potential to be treated by such methods. To explore the potential of this approach, we mated the conditional arginase deficient mouse with a mouse model deficient in fumarylacetoacetate hydrolase (FAH) and with Rag2 and IL2-Rγ mutations to give a selective advantage to transplanted (normal) human hepatocytes. On day -1, a uroplasminogen-expressing adenoviral vector was administered intravenously followed the next day with the transplantation of 1 × 10

Indexed as

Genetic Predisposition to DiseaseAnimalsArginaseCells, CulturedDisease Models, AnimalFemaleHepatocytesHumansLiver DiseasesMaleMetabolic DiseasesMiceMice, KnockoutArginaseAmmoniaArginase deficiencyCellular transplantHyperargininemiaTreatmentUrea cycle disorder

Identifiers

PMID29724658
PMCPMC5976549
OpenAlexW2799462699

What OpenQuestion holds

Textmetadata
LicenceTDM
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.