Uncovering cell type-specific phenotypes using a novel human in vitro model of transthyretin amyloid cardiomyopathy

Summary

Our model shows that WT and variant TTR fibrils lead to cell type-specific phenotypes, providing novel insights into the underlying cellular disease mechanisms of ATTR-CM, thereby facilitating the identification of novel therapeutic targets and biomarkers. © 2025. The Author(s).

Authors Qin J, Yang Q, Ullate-Agote A, Sampaio-Pinto V, Florit L, Dokter I, Mathioudaki C, Middelberg L, Montero-Calle P, Aguirre-Ruiz P, de Las Heras Rojo J, Lei Z, Qiu Z, Wei J, van der Harst P, Prosper F, Mazo MM, Iglesias-García O, Minnema MC, Sluijter JPG, Oerlemans MIFJ, van Mil A
Journal Stem cell research & therapy
Publication Date 2025 Jul 6;16(1):352
PubMed 40619379
PubMed Central PMC12232587
DOI 10.1186/s13287-025-04464-6

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