Evidence map›Paper›PMID 31890731›Full record

ArticleMolecular therapy. Methods & clinical development2019

Pathogenesis of Hepatic Tumors following Gene Therapy in Murine and Canine Models of Glycogen Storage Disease.

Hye-Ri Kang, Monika Gjorgjieva, Stephanie N Smith, Elizabeth D Brooks, Zelin Chen, Shawn M Burgess, Randy J Chandler, Lauren R Waskowicz, Kylie M Grady, Songtao Li and 4 more

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

Article in Molecular therapy. Methods & clinical development, 2019. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 8 papers.

0numbers the graph read from it
0cells of the map it votes in
8citing papers in PubMed
0.7field-weighted citation impact, top 26% 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

8 citing papers in PubMed, 17 citations in OpenAlex.

  1. Review
  2. Rescue of neurologic disease in mucopolysaccharidosis type II mice via AAV-mediated liver delivery of brain-penetrating iduronate-2-sulfatase.Neurotherapeutics : the journal of the American Society for Experimental NeuroTherapeutics · 2025
    Article
  3. Review
  4. Gene therapy for glycogen storage diseases.Journal of inherited metabolic disease · 2024
    Review
  5. Review
  6. Gene therapy for liver diseases - progress and challenges.Nature reviews. Gastroenterology & hepatology · 2023
    Review
  7. Article
  8. Evaluating the state of the science for adeno-associated virus integration: An integrated perspective.Molecular therapy : the journal of the American Society of Gene Therapy · 2022
    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

14 authors at 3 institutions in 2 countries.

Hye-Ri KangDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Monika GjorgjievaU1213, Institut National de la Santé et de la Recherche Médicale, Université Lyon 1, Lyon, France.
Stephanie N SmithDevelopmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Elizabeth D BrooksDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Zelin ChenOrganic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Shawn M BurgessOrganic Acid Research Section, Medical Genomics and Metabolic Genetics Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Randy J ChandlerDevelopmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Lauren R WaskowiczDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Kylie M GradyDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Songtao LiDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Gilles MithieuxU1213, Institut National de la Santé et de la Recherche Médicale, Université Lyon 1, Lyon, France.
Charles P VendittiDevelopmental Genomics Section, Translational and Functional Genomics Branch, National Human Genome Research Institute, Bethesda, MD, USA.
Fabienne RajasU1213, Institut National de la Santé et de la Recherche Médicale, Université Lyon 1, Lyon, France.
Dwight D KoeberlDivision of Medical Genetics, Department of Pediatrics, Duke University School of Medicine, Durham, NC, USA.
Duke University · USNational Human Genome Research Institute · USUniversité Claude Bernard Lyon 1 · FR

Funding

Investigating Autophagy in GSD-IaR01DK105434 · NIDDK · DUKE UNIVERSITY · PI KOEBERL, DWIGHT D · 2016 to 2019
$1.3M
NIDDK NIH HHS R01 DK105434
6 · The paper itself

Abstract

Glycogen storage disease type Ia (GSD Ia) is caused by mutations in the glucose-6-phosphatase (G6Pase) catalytic subunit gene (

Indexed as

genome editingglucose-6-phosphataseglycogen storage disorder type Iahepatocellular carcinomavon Gierke disease

Identifiers

PMID31890731
PMCPMC6909089
OpenAlexW2986855929

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
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.