Generation and characterization of induced pluripotent stem cell (iPSC) lines from two symptomatic carriers of pathogenic TNNI3 variants and two non-carrier relatives

Pediatric restrictive cardiomyopathy (RCM) is a rare, life-threatening heart disorder characterized by myocardial stiffness and impaired ventricular relaxation. It is often linked to pathogenic TNNI3 variants, which affect cardiac troponin I, a key regulator of myocardial contraction and relaxation. We generated induced pluripotent stem cell (iPSC) lines from two affected children, carrying the c.574C > T(p.Arg192Cys) and c.509G > A (p.Arg170Gln) variants, and from two unaffected family members. All iPSC lines exhibited typical morphology, expressed markers of undifferentiated state, and demonstrated multipotent differentiation potential. These patient-specific lines provide a physiologically relevant platform to investigate disease mechanisms and develop targeted therapies for TNNI3-associated RCM.

Authors

Institutions

Publication Details

Journal
Stem Cell Research
Published
2026-03-05
DOI
https://doi.org/10.1016/j.scr.2026.103961
Primary Topic
Genomics and Rare Diseases
Type
article
Field-Weighted Citation Impact
0.00
Controls
|||
ALL TIME
JAN
FEB
MAR
APR
MAY
JUN
JUL
AUG
SEP
OCT
article

Generation and characterization of induced pluripotent stem cell (iPSC) lines from two symptomatic carriers of pathogenic TNNI3 variants and two non-carrier relatives

Beatrijs Bartelds, Francesca Stillitano, Henk S. Schipper, Femke Pol et al.
Stem Cell Research
Genomics and Rare Diseases
article

Generation and characterization of induced pluripotent stem cell (iPSC) lines from two symptomatic carriers of pathogenic TNNI3 variants and two non-carrier relatives

Beatrijs Bartelds, Francesca Stillitano, Henk S. Schipper, Femke Pol, Pieter Doevendans, Michele F. Buono, Paulo Starčević
article en

Abstract

Pediatric restrictive cardiomyopathy (RCM) is a rare, life-threatening heart disorder characterized by myocardial stiffness and impaired ventricular relaxation. It is often linked to pathogenic TNNI3 variants, which affect cardiac troponin I, a key regulator of myocardial contraction and relaxation. We generated induced pluripotent stem cell (iPSC) lines from two affected children, carrying the c.574C > T(p.Arg192Cys) and c.509G > A (p.Arg170Gln) variants, and from two unaffected family members. All iPSC lines exhibited typical morphology, expressed markers of undifferentiated state, and demonstrated multipotent differentiation potential. These patient-specific lines provide a physiologically relevant platform to investigate disease mechanisms and develop targeted therapies for TNNI3-associated RCM.

Stem Cell ResearchVol. 93
Utrecht University (NL), University Medical Center Utrecht (NL), Netherlands Heart Institute (NL), Erasmus MC - Sophia Children’s Hospital (NL)
Zero hunger
Openalex Percentile: Top 35%
Genomics and Rare Diseases
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.