Evidence map›Paper›PMID 40762880›Full record

ArticleStem cell reviews and reports2025

Granulopoietic Dysregulation in a Patient-Tailored Mouse Model of Barth Syndrome.

Elizabeth A Sierra Potchanant, Maegan L Capitano, Donna M Edwards, Baskar Ramdas, Scott Cooper, James Ropa, S Louise Pay, Aditya Sheth, Paige L Snider, Hilary J Vernon and 3 more

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In one paragraph

Article in Stem cell reviews and reports, 2025. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. 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. Article
  2. Article
  3. Article
4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

13 authors.

Elizabeth A Sierra Potchanant *Department of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Maegan L Capitano *Department of Microbiology & Immunology, Indiana University School of Medicine, 635 Barnhill Drive, Indianapolis, IN, 46202, USA. malcapit@iu.edu.
Donna M EdwardsDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Baskar RamdasDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Scott CooperDepartment of Microbiology & Immunology, Indiana University School of Medicine, 635 Barnhill Drive, Indianapolis, IN, 46202, USA.
James RopaDepartment of Medical & Molecular Genetics, Indiana University School of Medicine, 410 West 10th Street, Indianapolis, IN, 46202, USA.
S Louise PayStark Neurosciences Research Institute, Indiana University School of Medicine, 320 West 15th Street, Indianapolis, IN, 46202, USA.
Aditya ShethDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Paige L SniderDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Hilary J VernonDepartment of Genetic Medicine, Johns Hopkins University School of Medicine, 733 North Broadway, Baltimore, MD, 21205, USA.
Ngoc-Tung TranDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Reuben KapurDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA.
Simon J ConwayDepartment of Pediatrics, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, 1044 West Walnut Street, Indianapolis, IN, 46202, USA. siconway@iu.edu.

Funding

Project-005U54DK106846 · NIDDK · INDIANA UNIVERSITY INDIANAPOLIS · PI Reuben Kapur, Karen Elizabeth Pollok · 2015 to 2026
$9.7M
Tafazzin and metabolic reprogramming during cardiomyopathyR01HL159436 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI CONWAY, SIMON JAMES · 2021 to 2024
$2.3M
Omics interrogation of functionally competent hematopoieitic stem cellsR00HL166790 · NHLBI · INDIANA UNIVERSITY INDIANAPOLIS · PI James Patrick Ropa · 2025 to 2026
$497k
NHLBI NIH HHS R00 HL166790NHLBI NIH HHS R01 HL159436NIDDK NIH HHS U54 DK106846NIH HHS R01 HL159436NIH HHS U54 DK106846
6 · The paper itself

Abstract

Barth syndrome (BTHS) is an X-linked recessive disorder characterized by cardiomyopathy, skeletal muscle myopathy and fatigue, growth restriction, and neutropenia. Neutropenia increases the risk of life-threatening bacterial infections, a major cause of death in individuals with BTHS. Currently, there is no curative treatment for BTHS or associated neutropenia. The development of therapeutic strategies to correct BTHS-associated neutropenia has been hindered by a limited understanding of the underlying molecular mechanisms involved. BTHS is caused by a mutation in the Tafazzin gene encoding a transacylase required for the maturation of cardiolipin, an inner mitochondrial membrane phospholipid crucial for mitochondrial structure and function. We introduced a BTHS patient's point mutation (TAZ

Indexed as

Barth SyndromeTranscription FactorsAcyltransferasesAnimalsDisease Models, AnimalHumansMaleMiceMitochondriaNeutropeniaNeutrophilsAcyltransferasestafazzin protein, mouseTranscription FactorsBarth syndromeHematopoietic stem cellsNeutropeniaPatient-tailored mouse modelTafazzin

Identifiers

PMID40762880
PMCPMC12408712

What Socratic 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 Socratic graph.