Cardiac MRI reveals myocardial fibrosis and systolic dysfunction in mitochondrial trifunctional protein–deficient mice

Cardiomyopathy is an important manifestation in patients with fatty acid oxidation disorders and represents a major cause of morbidity and early mortality in mitochondrial trifunctional protein (TFP) deficiency. Although a mouse model carrying the TFP β-subunit p.Met404Lys mutation (βTFP-deficient) has been described, cardiac involvement in this model has not been systematically characterized. Here, we combined cardiac histology and multiparametric cardiac MRI (CMR) to define myocardial structure, function, and tissue characteristics in this mouse model. Histological analysis with automated whole-slide collagen quantification revealed myocardial fibrosis with collagen deposition in mutant hearts, and CMR demonstrated increased myocardial extracellular volume in both male and female homozygous mutants. Homozygous males showed reduced ejection fraction, impaired systolic strain, and increased left ventricular end-systolic volume, indicating systolic dysfunction. Male mice were more severely affected than females and exhibited reduced survival. Together, these findings demonstrate that βTFP-deficient mice develop fibrotic cardiomyopathy with systolic dysfunction, reproducing important cardiac features observed in human TFP deficiency. This work establishes the model as a relevant platform for investigating disease mechanisms and therapeutic strategies for cardiomyopathy in TFP deficiency.

Authors

Institutions

Publication Details

Journal
Disease Models & Mechanisms
Published
2026-08-03
DOI
https://doi.org/10.1242/dmm.052921
Primary Topic
Mitochondrial Function and Pathology
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Cardiac MRI reveals myocardial fibrosis and systolic dysfunction in mitochondrial trifunctional protein–deficient mice

Eduardo Vieira Neto, Yijen Wu, Shakuntala Basu, Jerry E. Vockley et al.
Disease Models & Mechanisms
Mitochondrial Function and Pathology
article

Cardiac MRI reveals myocardial fibrosis and systolic dysfunction in mitochondrial trifunctional protein–deficient mice

Eduardo Vieira Neto, Yijen Wu, Shakuntala Basu, Jerry E. Vockley, Sean Hartwick, Kristina Schwab, Thomas J. Becker-Szurszewski
article en

Abstract

Cardiomyopathy is an important manifestation in patients with fatty acid oxidation disorders and represents a major cause of morbidity and early mortality in mitochondrial trifunctional protein (TFP) deficiency. Although a mouse model carrying the TFP β-subunit p.Met404Lys mutation (βTFP-deficient) has been described, cardiac involvement in this model has not been systematically characterized. Here, we combined cardiac histology and multiparametric cardiac MRI (CMR) to define myocardial structure, function, and tissue characteristics in this mouse model. Histological analysis with automated whole-slide collagen quantification revealed myocardial fibrosis with collagen deposition in mutant hearts, and CMR demonstrated increased myocardial extracellular volume in both male and female homozygous mutants. Homozygous males showed reduced ejection fraction, impaired systolic strain, and increased left ventricular end-systolic volume, indicating systolic dysfunction. Male mice were more severely affected than females and exhibited reduced survival. Together, these findings demonstrate that βTFP-deficient mice develop fibrotic cardiomyopathy with systolic dysfunction, reproducing important cardiac features observed in human TFP deficiency. This work establishes the model as a relevant platform for investigating disease mechanisms and therapeutic strategies for cardiomyopathy in TFP deficiency.

Disease Models & Mechanisms
University of Pittsburgh (US), Children's Institute of Pittsburgh (US), Children's Hospital of Pittsburgh (US)
Good health and well-being
Openalex Percentile: Top 16%
Mitochondrial Function and Pathology
AI Navigator

Ask Laika to Summarize, Analyze, and Connect papers live on the map.

Summarize Papers & Methodologies

Extract key findings, datasets, and comparative methods across publications.

Benchmark Rankings & Visual Analytics

Rank top research institutions, authors, funders, topics, and journals by Field-Weighted Citation Impact (FWCI) and paper volume with instant charts.

Connect Distant Disciplines

Bridge topological clusters on the map to find hidden collaborative intersections.