Evidence map›Paper›PMID 39488079›Full record

ArticleMolecular genetics and metabolism2024

Progressive liver disease and dysregulated glycogen metabolism in murine GSD IX γ2 models human disease.

Rebecca A Gibson, William R Jeck, Rebecca L Koch, Aarav Mehta, Su Jin Choi, Yajur Sriraman, Deeksha Bali, Sarah Young, Aravind Asokan, Jeong-A Lim and 1 more

Abstract read
PubMed Full text at PMC Publisher
In one paragraph

Article in Molecular genetics and metabolism, 2024. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 3 papers.

0numbers the graph read from it
0cells of the map it votes in
3citing 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

3 citing papers in PubMed.

  1. Observational
  2. Article
  3. 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.

Rebecca A GibsonDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, USA; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
William R JeckDepartment of Pathology, Duke University Medical Center, Durham, NC, USA.
Rebecca L KochDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Aarav MehtaDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Su Jin ChoiDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Yajur SriramanDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Deeksha BaliDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Sarah YoungDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Aravind AsokanDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, USA; Department of Surgery, Duke University Medical Center, Durham, NC, USA.
Jeong-A LimDivision of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA.
Priya S KishnaniDepartment of Molecular Genetics and Microbiology, Duke University Medical Center, Durham, NC, USA; Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NC, USA. Electronic address: priya.kishnani@duke.edu.

Funding

Liver-directed AAV gene therapy for PHKG2-Glycogen Storage Disease IX (GSD IX y2)F30HD104480 · NICHD · DUKE UNIVERSITY · PI GIBSON, REBECCA ANNE · 2021 to 2023
$121k
NICHD NIH HHS F30 HD104480
6 · The paper itself

Abstract

Hepatic glycogen storage disease type IX γ2 (GSD IX γ2) is a severe, liver-specific subtype of GSD IX. While all patients with hepatic GSD IX present with similar symptoms, over 95 % of patients with GSD IX γ2 progress to liver fibrosis and cirrhosis. Despite disease severity, the long-term natural history of GSD IX γ2 liver disease progression is not known. Our lab previously characterized the Phkg2

Indexed as

Disease Models, AnimalDisease ProgressionGlycogenGlycogen Storage DiseaseLiverLiver CirrhosisAnimalsBiomarkersHumansLiver DiseasesMaleMiceMice, KnockoutBiomarkersGlycogenGlycogenesisGlycogenolysisGlycogen storage disease type IXGSD IXcGSD IX gamma2GSD IX γ2Liver fibrosisMouse modelPhkg2Phosphorylase kinase deficiency

Identifiers

PMID39488079
PMCPMC11633833

What OpenQuestion holds

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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.