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
- Beatrijs Bartelds (ORCID: https://orcid.org/0000-0003-3151-2474)
- Francesca Stillitano (ORCID: https://orcid.org/0000-0003-1898-5058)
- Henk S. Schipper (ORCID: https://orcid.org/0000-0003-1997-8060)
- Femke Pol
- Pieter Doevendans
- Michele F. Buono
- Paulo Starčević
Institutions
- Utrecht University (NL)
- University Medical Center Utrecht (NL)
- Netherlands Heart Institute (NL)
- Erasmus MC - Sophia Children’s Hospital (NL)
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