{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,10]],"date-time":"2026-11-10T03:51:03Z","timestamp":1794282663687,"version":"4.9.0"},"reference-count":58,"publisher":"Springer Science and Business Media LLC","issue":"1","license":[{"start":{"date-parts":[[2025,7,6]],"date-time":"2025-07-06T00:00:00Z","timestamp":1751760000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0"},{"start":{"date-parts":[[2025,7,6]],"date-time":"2025-07-06T00:00:00Z","timestamp":1751760000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0"}],"funder":[{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","award":["67570063"],"award-info":[{"award-number":["67570063"]}],"id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100004319","name":"Pfizer","doi-asserted-by":"publisher","award":["67562235"],"award-info":[{"award-number":["67562235"]}],"id":[{"id":"10.13039\/100004319","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100014470","name":"Netherlands Heart Institute","doi-asserted-by":"publisher","award":["Netherlands Heart Institute"],"award-info":[{"award-number":["Netherlands Heart Institute"]}],"id":[{"id":"10.13039\/501100014470","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010663","name":"H2020 European Research Council","doi-asserted-by":"publisher","award":["874827"],"award-info":[{"award-number":["874827"]}],"id":[{"id":"10.13039\/100010663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010663","name":"H2020 European Research Council","doi-asserted-by":"publisher","award":["874827"],"award-info":[{"award-number":["874827"]}],"id":[{"id":"10.13039\/100010663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010663","name":"H2020 European Research Council","doi-asserted-by":"publisher","award":["874827"],"award-info":[{"award-number":["874827"]}],"id":[{"id":"10.13039\/100010663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010663","name":"H2020 European Research Council","doi-asserted-by":"publisher","award":["874827"],"award-info":[{"award-number":["874827"]}],"id":[{"id":"10.13039\/100010663","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100017266","name":"Gobierno de Navarra","doi-asserted-by":"publisher","award":["0011-1411-2022-000092"],"award-info":[{"award-number":["0011-1411-2022-000092"]}],"id":[{"id":"10.13039\/501100017266","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100017266","name":"Gobierno de Navarra","doi-asserted-by":"publisher","award":["PC020-021-022"],"award-info":[{"award-number":["PC020-021-022"]}],"id":[{"id":"10.13039\/501100017266","id-type":"DOI","asserted-by":"publisher"}]},{"name":"INVESTTRA","award":["PID2022-142807OA-I00"],"award-info":[{"award-number":["PID2022-142807OA-I00"]}]},{"name":"Sara Borrell grant","award":["CD22\/00027"],"award-info":[{"award-number":["CD22\/00027"]}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Stem Cell Res Ther"],"DOI":"10.1186\/s13287-025-04464-6","type":"journal-article","created":{"date-parts":[[2025,7,6]],"date-time":"2025-07-06T14:21:13Z","timestamp":1751811673000},"update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Uncovering cell type-specific phenotypes using a novel human in vitro model of transthyretin amyloid cardiomyopathy"],"prefix":"10.1186","volume":"16","author":[{"given":"Jiabin","family":"Qin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiangbing","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Asier","family":"Ullate-Agote","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vasco","family":"Sampaio-Pinto","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Florit","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Inge","family":"Dokter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chrysoula","family":"Mathioudaki","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lotte","family":"Middelberg","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Montero-Calle","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paula","family":"Aguirre-Ruiz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joana","family":"de las Heras Rojo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhiyong","family":"Lei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zeping","family":"Qiu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jin","family":"Wei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pim","family":"van der Harst","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Felipe","family":"Prosper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manuel M.","family":"Mazo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olalla","family":"Iglesias-Garc\u00eda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monique C.","family":"Minnema","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joost P. G.","family":"Sluijter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3166-518X","authenticated-orcid":false,"given":"Marish I.F.J.","family":"Oerlemans","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alain","family":"van Mil","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2025,7,6]]},"reference":[{"issue":"9","key":"4464_CR1","doi-asserted-by":"publisher","first-page":"778","DOI":"10.1001\/jama.2024.0442","volume":"331","author":"FL Ruberg","year":"2024","unstructured":"Ruberg FL, Maurer MS. Cardiac amyloidosis due to transthyretin protein: a review. JAMA. 2024;331(9):778\u201391.","journal-title":"JAMA"},{"issue":"4","key":"4464_CR2","doi-asserted-by":"publisher","first-page":"213","DOI":"10.1080\/13506129.2022.2147636","volume":"29","author":"JN Buxbaum","year":"2022","unstructured":"Buxbaum JN, Dispenzieri A, Eisenberg DS, F\u00e4ndrich M, Merlini G, Saraiva MJM, et al. Amyloid nomenclature 2022: update, novel proteins, and recommendations by the international society of amyloidosis (ISA) nomenclature committee. Amyloid. 2022;29(4):213\u20139.","journal-title":"Amyloid"},{"key":"4464_CR3","doi-asserted-by":"crossref","unstructured":"Muller SA, Peir\u00f3-Aventin B, Biagioni G, Tini G, Saturi G, Kronberger C et al. Evaluation of the 2021 ESC recommendations for family screening in hereditary transthyretin cardiac amyloidosis. Eur J Heart Fail. 2024. Sep;26(9):2025-2034","DOI":"10.1093\/eurheartj\/ehae666.1985"},{"issue":"7","key":"4464_CR4","doi-asserted-by":"publisher","first-page":"733","DOI":"10.1038\/gim.2016.200","volume":"19","author":"JN Buxbaum","year":"2017","unstructured":"Buxbaum JN, Ruberg FL. Transthyretin V122I (pV142I)* cardiac amyloidosis: an age-dependent autosomal dominant cardiomyopathy too common to be overlooked as a cause of significant heart disease in elderly African Americans. Genet Med. 2017;19(7):733\u201342.","journal-title":"Genet Med"},{"issue":"11","key":"4464_CR5","doi-asserted-by":"publisher","first-page":"525","DOI":"10.1007\/s12471-019-1299-1","volume":"27","author":"MIFJ Oerlemans","year":"2019","unstructured":"Oerlemans MIFJ, Rutten KHG, Minnema MC, Raymakers RAP, Asselbergs FW, de Jonge N. Cardiac amyloidosis: the need for early diagnosis. Neth Heart J. 2019;27(11):525\u201336.","journal-title":"Neth Heart J"},{"issue":"4","key":"4464_CR6","doi-asserted-by":"publisher","first-page":"512","DOI":"10.1002\/ejhf.2140","volume":"23","author":"P Garcia-Pavia","year":"2021","unstructured":"Garcia-Pavia P, Rapezzi C, Adler Y, Arad M, Basso C, Brucato A, et al. Diagnosis and treatment of cardiac amyloidosis. A position statement of the European society of cardiology working group on myocardial and pericardial diseases. Eur J Heart Fail. 2021;23(4):512\u201326.","journal-title":"Eur J Heart Fail"},{"issue":"2","key":"4464_CR7","doi-asserted-by":"publisher","first-page":"oeac011","DOI":"10.1093\/ehjopen\/oeac011","volume":"2","author":"M Brons","year":"2022","unstructured":"Brons M, Muller SA, Rutten FH, van der Meer MG, Vrancken AFJE, Minnema MC, et al. Evaluation of the cardiac amyloidosis clinical pathway implementation: a real-world experience. Eur Heart J Open. 2022;2(2):oeac011.","journal-title":"Eur Heart J Open"},{"issue":"10","key":"4464_CR8","doi-asserted-by":"publisher","first-page":"655","DOI":"10.1038\/s41569-022-00683-z","volume":"19","author":"A Aimo","year":"2022","unstructured":"Aimo A, Castiglione V, Rapezzi C, Franzini M, Panichella G, Vergaro G, et al. RNA-targeting and gene editing therapies for transthyretin amyloidosis. Nat Rev Cardiol. 2022;19(10):655\u201367.","journal-title":"Nat Rev Cardiol"},{"issue":"11","key":"4464_CR9","doi-asserted-by":"publisher","first-page":"1007","DOI":"10.1056\/NEJMoa1805689","volume":"379","author":"MS Maurer","year":"2018","unstructured":"Maurer MS, Schwartz JH, Gundapaneni B, Elliott PM, Merlini G, Waddington-Cruz M, et al. Tafamidis treatment for patients with transthyretin amyloid cardiomyopathy. N Engl J Med. 2018;379(11):1007\u201316.","journal-title":"N Engl J Med"},{"issue":"2","key":"4464_CR10","doi-asserted-by":"publisher","first-page":"115","DOI":"10.1016\/j.jchf.2020.09.011","volume":"9","author":"C Rapezzi","year":"2021","unstructured":"Rapezzi C, Elliott P, Damy T, Nativi-Nicolau J, Berk JL, Velazquez EJ, et al. Efficacy of tafamidis in patients with hereditary and wild-type transthyretin amyloid cardiomyopathy: further analyses from ATTR-ACT. JACC Heart Fail. 2021;9(2):115\u201323.","journal-title":"JACC Heart Fail"},{"issue":"2","key":"4464_CR11","doi-asserted-by":"publisher","first-page":"132","DOI":"10.1056\/NEJMoa2305434","volume":"390","author":"JD Gillmore","year":"2024","unstructured":"Gillmore JD, Judge DP, Cappelli F, Fontana M, Garcia-Pavia P, Gibbs S, et al. Efficacy and safety of acoramidis in transthyretin amyloid cardiomyopathy. N Engl J Med. 2024;390(2):132\u201342.","journal-title":"N Engl J Med"},{"issue":"1","key":"4464_CR12","doi-asserted-by":"publisher","first-page":"11","DOI":"10.1056\/NEJMoa1716153","volume":"379","author":"D Adams","year":"2018","unstructured":"Adams D, Gonzalez-Duarte A, O\u2019Riordan WD, Yang CC, Ueda M, Kristen AV, et al. Patisiran, an RNAi therapeutic, for hereditary transthyretin amyloidosis. N Engl J Med. 2018;379(1):11\u201321.","journal-title":"N Engl J Med"},{"issue":"6","key":"4464_CR13","doi-asserted-by":"publisher","first-page":"493","DOI":"10.1056\/NEJMoa2107454","volume":"385","author":"JD Gillmore","year":"2021","unstructured":"Gillmore JD, Gane E, Taubel J, Kao J, Fontana M, Maitland ML, et al. CRISPR-Cas9 in vivo gene editing for transthyretin amyloidosis. N Engl J Med. 2021;385(6):493\u2013502.","journal-title":"N Engl J Med"},{"issue":"17","key":"4464_CR14","doi-asserted-by":"publisher","first-page":"1553","DOI":"10.1056\/NEJMoa2300757","volume":"389","author":"MS Maurer","year":"2023","unstructured":"Maurer MS, Kale P, Fontana M, Berk JL, Grogan M, Gustafsson F, et al. Patisiran treatment in patients with transthyretin cardiac amyloidosis. N Engl J Med. 2023;389(17):1553\u201365.","journal-title":"N Engl J Med"},{"issue":"3","key":"4464_CR15","doi-asserted-by":"publisher","first-page":"239","DOI":"10.1056\/NEJMoa2303765","volume":"389","author":"P Garcia-Pavia","year":"2023","unstructured":"Garcia-Pavia P, Aus dem Siepen F, Donal E, Lairez O, van der Meer P, Kristen AV, et al. Phase 1 trial of antibody NI006 for depletion of cardiac transthyretin amyloid. N Engl J Med. 2023;389(3):239\u201350.","journal-title":"N Engl J Med"},{"issue":"30","key":"4464_CR16","doi-asserted-by":"publisher","first-page":"10446","DOI":"10.1074\/jbc.RA120.013135","volume":"295","author":"KW Walker","year":"2020","unstructured":"Walker KW, Foltz IN, Wang T, Salimi-Moosavi H, Bailis JM, Lee F, et al. The serum protein transthyretin as a platform for dimerization and tetramerization of antibodies and fab fragments to enable target clustering. J Biol Chem. 2020;295(30):10446\u201355.","journal-title":"J Biol Chem"},{"issue":"1","key":"4464_CR17","doi-asserted-by":"publisher","first-page":"55","DOI":"10.1016\/S1046-5928(03)00069-X","volume":"30","author":"K Matsubara","year":"2003","unstructured":"Matsubara K, Mizuguchi M, Kawano K. Expression of a synthetic gene encoding human transthyretin in Escherichia coli. Protein Expr Purif. 2003;30(1):55\u201361.","journal-title":"Protein Expr Purif"},{"issue":"48","key":"4464_CR18","doi-asserted-by":"publisher","first-page":"28932","DOI":"10.1074\/jbc.M115.659912","volume":"290","author":"L Saelices","year":"2015","unstructured":"Saelices L, Johnson LM, Liang WY, Sawaya MR, Cascio D, Ruchala P, et al. Uncovering the mechanism of aggregation of human transthyretin. J Biol Chem. 2015;290(48):28932\u201343.","journal-title":"J Biol Chem"},{"issue":"23","key":"4464_CR19","doi-asserted-by":"publisher","first-page":"7365","DOI":"10.1021\/bi049621l","volume":"43","author":"AR Hurshman","year":"2004","unstructured":"Hurshman AR, White JT, Powers ET, Kelly JW. Transthyretin aggregation under partially denaturing conditions is a downhill polymerization. Biochemistry. 2004;43(23):7365\u201381.","journal-title":"Biochemistry"},{"issue":"1","key":"4464_CR20","doi-asserted-by":"publisher","first-page":"160696","DOI":"10.1098\/rsos.160696","volume":"4","author":"C Xue","year":"2017","unstructured":"Xue C, Lin TY, Chang D, Guo Z. Thioflavin T as an amyloid dye: fibril quantification, optimal concentration and effect on aggregation. R Soc Open Sci. 2017;4(1):160696.","journal-title":"R Soc Open Sci"},{"issue":"24","key":"4464_CR21","doi-asserted-by":"publisher","first-page":"7222","DOI":"10.7150\/thno.32058","volume":"9","author":"S Hamad","year":"2019","unstructured":"Hamad S, Derichsweiler D, Papadopoulos S, Nguemo F, \u0160ari\u0107 T, Sachinidis A, et al. Generation of human induced pluripotent stem cell-derived cardiomyocytes in 2D monolayer and scalable 3D suspension bioreactor cultures with reduced batch-to-batch variations. Theranostics. 2019;9(24):7222\u201338.","journal-title":"Theranostics"},{"issue":"1","key":"4464_CR22","doi-asserted-by":"publisher","first-page":"50","DOI":"10.1016\/j.stem.2020.06.001","volume":"27","author":"JW Buikema","year":"2020","unstructured":"Buikema JW, Lee S, Goodyer WR, Maas RG, Chirikian O, Li G, et al. Wnt activation and reduced cell-cell contact synergistically induce massive expansion of functional human iPSC-derived cardiomyocytes. Cell Stem Cell. 2020;27(1):50\u2013e635.","journal-title":"Cell Stem Cell"},{"issue":"1","key":"4464_CR23","doi-asserted-by":"publisher","first-page":"100334","DOI":"10.1016\/j.xpro.2021.100334","volume":"2","author":"RGC Maas","year":"2021","unstructured":"Maas RGC, Lee S, Harakalova M, Snijders Blok CJB, Goodyer WR, Hjortnaes J, et al. Massive expansion and cryopreservation of functional human induced pluripotent stem cell-derived cardiomyocytes. STAR Protocols. 2021;2(1):100334.","journal-title":"STAR Protocols"},{"key":"4464_CR24","doi-asserted-by":"publisher","first-page":"1015473","DOI":"10.3389\/fcvm.2022.1015473","volume":"9","author":"MR Moita","year":"2022","unstructured":"Moita MR, Silva MM, Diniz C, Serra M, Hoet RM, Barbas A, et al. Transcriptome and proteome profiling of activated cardiac fibroblasts supports target prioritization in cardiac fibrosis. Front Cardiovasc Med. 2022;9:1015473.","journal-title":"Front Cardiovasc Med"},{"issue":"5","key":"4464_CR25","doi-asserted-by":"publisher","first-page":"e15207","DOI":"10.14814\/phy2.15207","volume":"10","author":"KT Dittloff","year":"2022","unstructured":"Dittloff KT, Spanghero E, Sol\u00eds C, Banach K, Russell B. Transthyretin deposition alters cardiomyocyte sarcomeric architecture, calcium transients, and contractile force. Physiol Rep. 2022;10(5):e15207.","journal-title":"Physiol Rep"},{"issue":"1","key":"4464_CR26","doi-asserted-by":"publisher","first-page":"H149","DOI":"10.1152\/ajpheart.00073.2021","volume":"321","author":"KT Dittloff","year":"2021","unstructured":"Dittloff KT, Iezzi A, Zhong JX, Mohindra P, Desai TA, Russell B. Transthyretin amyloid fibrils alter primary fibroblast structure, function, and inflammatory gene expression. Am J Physiol Heart Circ Physiol. 2021;321(1):H149\u201360.","journal-title":"Am J Physiol Heart Circ Physiol"},{"key":"4464_CR27","doi-asserted-by":"crossref","unstructured":"Ainsworth MJ, Chirico N, de Ruijter M, Hrynevich A, Dokter I, Sluijter JPG et al. Convergence of melt electrowriting and extrusion-based bioprinting for vascular patterning of a myocardial construct. Biofabrication. 2023;15(3).","DOI":"10.1088\/1758-5090\/ace07f"},{"issue":"15","key":"4464_CR28","doi-asserted-by":"publisher","first-page":"3866","DOI":"10.1039\/D3BM01908J","volume":"12","author":"J Janssen","year":"2024","unstructured":"Janssen J, Chirico N, Ainsworth MJ, Cedillo-Servin G, Viola M, Dokter I, et al. Hypothermic and cryogenic preservation of cardiac tissue-engineered constructs. Biomater Sci. 2024;12(15):3866\u201381.","journal-title":"Biomater Sci"},{"issue":"1","key":"4464_CR29","doi-asserted-by":"publisher","first-page":"531","DOI":"10.1186\/s13287-022-03209-z","volume":"13","author":"N Chirico","year":"2022","unstructured":"Chirico N, Kessler EL, Maas RGC, Fang J, Qin J, Dokter I, et al. Small molecule-mediated rapid maturation of human induced pluripotent stem cell-derived cardiomyocytes. Stem Cell Res Ther. 2022;13(1):531.","journal-title":"Stem Cell Res Ther"},{"key":"4464_CR30","unstructured":"De Boer T. Peaks, software for analysing cardiac APs and calcium transients. Open Sci Framew. 2019."},{"issue":"1","key":"4464_CR31","doi-asserted-by":"publisher","first-page":"800","DOI":"10.1038\/s42003-023-05165-7","volume":"6","author":"MT Roefs","year":"2023","unstructured":"Roefs MT, Bauz\u00e1-Martinez J, van de Wakker SI, Qin J, Olijve WT, Tuinte R, et al. Cardiac progenitor cell-derived extracellular vesicles promote angiogenesis through both associated- and co-isolated proteins. Commun Biol. 2023;6(1):800.","journal-title":"Commun Biol"},{"issue":"7","key":"4464_CR32","doi-asserted-by":"publisher","first-page":"e0232565","DOI":"10.1371\/journal.pone.0232565","volume":"15","author":"A Suarez-Arnedo","year":"2020","unstructured":"Suarez-Arnedo A, Torres Figueroa F, Clavijo C, Arbel\u00e1ez P, Cruz JC, Mu\u00f1oz-Camargo C. An image J plugin for the high throughput image analysis of in vitro scratch wound healing assays. PLoS ONE. 2020;15(7):e0232565.","journal-title":"PLoS ONE"},{"issue":"9","key":"4464_CR33","doi-asserted-by":"publisher","first-page":"1118","DOI":"10.1016\/j.jacep.2020.04.019","volume":"6","author":"E Donnellan","year":"2020","unstructured":"Donnellan E, Wazni OM, Hanna M, Elshazly MB, Puri R, Saliba W, et al. Atrial fibrillation in transthyretin cardiac amyloidosis: predictors, prevalence, and efficacy of rhythm control strategies. JACC Clin Electrophysiol. 2020;6(9):1118\u201327.","journal-title":"JACC Clin Electrophysiol"},{"issue":"4","key":"4464_CR34","doi-asserted-by":"publisher","first-page":"358","DOI":"10.1016\/j.jchf.2014.03.009","volume":"2","author":"S Dorbala","year":"2014","unstructured":"Dorbala S, Vangala D, Bruyere J, Quarta C, Kruger J, Padera R, et al. Coronary microvascular dysfunction is related to abnormalities in myocardial structure and function in cardiac amyloidosis. JACC Heart Fail. 2014;2(4):358\u201367.","journal-title":"JACC Heart Fail"},{"issue":"12","key":"4464_CR35","doi-asserted-by":"publisher","first-page":"1260","DOI":"10.1093\/eurheartj\/ehz695","volume":"41","author":"J George","year":"2020","unstructured":"George J, Rappaport M, Shimoni S, Goland S, Voldarsky I, Fabricant Y, et al. A novel monoclonal antibody targeting aggregated transthyretin facilitates its removal and functional recovery in an experimental model. Eur Heart J. 2020;41(12):1260\u201370.","journal-title":"Eur Heart J"},{"key":"4464_CR36","doi-asserted-by":"publisher","first-page":"1144049","DOI":"10.3389\/fmolb.2023.1144049","volume":"10","author":"C Teixeira","year":"2023","unstructured":"Teixeira C, Martins HS, Saraiva MJ. Cellular environment of TTR deposits in an animal model of ATTR-cardiomyopathy. Front Mol Biosci. 2023;10:1144049.","journal-title":"Front Mol Biosci"},{"issue":"1","key":"4464_CR37","doi-asserted-by":"publisher","first-page":"7112","DOI":"10.1038\/s41467-021-27416-z","volume":"12","author":"I Slamova","year":"2021","unstructured":"Slamova I, Adib R, Ellmerich S, Golos MR, Gilbertson JA, Botcher N, et al. Plasmin activity promotes amyloid deposition in a transgenic model of human transthyretin amyloidosis. Nat Commun. 2021;12(1):7112.","journal-title":"Nat Commun"},{"issue":"4","key":"4464_CR38","doi-asserted-by":"publisher","first-page":"707","DOI":"10.1016\/j.bbrc.2011.04.133","volume":"410","author":"S Bourgault","year":"2011","unstructured":"Bourgault S, Choi S, Buxbaum JN, Kelly JW, Price JL, Reixach N. Mechanisms of transthyretin cardiomyocyte toxicity inhibition by resveratrol analogs. Biochem Biophys Res Commun. 2011;410(4):707\u201313.","journal-title":"Biochem Biophys Res Commun"},{"key":"4464_CR39","doi-asserted-by":"crossref","unstructured":"Manral P, Reixach N. Amyloidogenic and non-amyloidogenic transthyretin variants interact differently with human cardiomyocytes: insights into early events of non-fibrillar tissue damage. Biosci Rep. 2015;35(1).","DOI":"10.1042\/BSR20140155"},{"issue":"24","key":"4464_CR40","doi-asserted-by":"publisher","first-page":"9992","DOI":"10.1073\/pnas.1300761110","volume":"110","author":"SC Penchala","year":"2013","unstructured":"Penchala SC, Connelly S, Wang Y, Park MS, Zhao L, Baranczak A, et al. AG10 inhibits amyloidogenesis and cellular toxicity of the familial amyloid cardiomyopathy-associated V122I transthyretin. Proc Natl Acad Sci USA. 2013;110(24):9992\u20137.","journal-title":"Proc Natl Acad Sci USA"},{"key":"4464_CR41","doi-asserted-by":"publisher","first-page":"10787","DOI":"10.1038\/ncomms10787","volume":"7","author":"R Sant\u2019Anna","year":"2016","unstructured":"Sant\u2019Anna R, Gallego P, Robinson LZ, Pereira-Henriques A, Ferreira N, Pinheiro F, et al. Repositioning tolcapone as a potent inhibitor of transthyretin amyloidogenesis and associated cellular toxicity. Nat Commun. 2016;7:10787.","journal-title":"Nat Commun"},{"issue":"1","key":"4464_CR42","doi-asserted-by":"publisher","first-page":"12508","DOI":"10.1038\/s41598-018-30995-5","volume":"8","author":"D Ami","year":"2018","unstructured":"Ami D, Mereghetti P, Leri M, Giorgetti S, Natalello A, Doglia SM, et al. A FTIR microspectroscopy study of the structural and biochemical perturbations induced by natively folded and aggregated transthyretin in HL-1 cardiomyocytes. Sci Rep. 2018;8(1):12508.","journal-title":"Sci Rep"},{"issue":"1","key":"4464_CR43","doi-asserted-by":"publisher","first-page":"129453","DOI":"10.1016\/j.bbagen.2019.129453","volume":"1864","author":"M Leri","year":"2020","unstructured":"Leri M, Rebuzzini P, Caselli A, Luti S, Natalello A, Giorgetti S, et al. S-homocysteinylation effects on transthyretin: worsening of cardiomyopathy onset. Biochim Biophys Acta Gen Subj. 2020;1864(1):129453.","journal-title":"Biochim Biophys Acta Gen Subj"},{"issue":"9","key":"4464_CR44","doi-asserted-by":"publisher","first-page":"2024","DOI":"10.1016\/j.bpj.2016.09.010","volume":"111","author":"L Sartiani","year":"2016","unstructured":"Sartiani L, Bucciantini M, Spinelli V, Leri M, Natalello A, Nosi D, et al. Biochemical and electrophysiological modification of amyloid transthyretin on cardiomyocytes. Biophys J. 2016;111(9):2024\u201338.","journal-title":"Biophys J"},{"issue":"4","key":"4464_CR45","doi-asserted-by":"publisher","first-page":"379","DOI":"10.1080\/13506129.2023.2224494","volume":"30","author":"S Ghosh","year":"2023","unstructured":"Ghosh S, Villacorta-Martin C, Lindstrom-Vautrin J, Kenney D, Golden CS, Edwards CV, et al. Mapping cellular response to destabilized transthyretin reveals cell- and amyloidogenic protein-specific signatures. Amyloid. 2023;30(4):379\u201393.","journal-title":"Amyloid"},{"issue":"5","key":"4464_CR46","doi-asserted-by":"publisher","first-page":"451","DOI":"10.1016\/j.stemcr.2013.10.003","volume":"1","author":"A Leung","year":"2013","unstructured":"Leung A, Nah SK, Reid W, Ebata A, Koch CM, Monti S, et al. Induced pluripotent stem cell modeling of multisystemic, hereditary transthyretin amyloidosis. Stem Cell Rep. 2013;1(5):451\u201363.","journal-title":"Stem Cell Rep"},{"issue":"3","key":"4464_CR47","doi-asserted-by":"publisher","first-page":"148","DOI":"10.1080\/13506129.2018.1489228","volume":"25","author":"RM Giadone","year":"2018","unstructured":"Giadone RM, Rosarda JD, Akepati PR, Thomas AC, Boldbaatar B, James MF, et al. A library of ATTR amyloidosis patient-specific induced pluripotent stem cells for disease modelling and in vitro testing of novel therapeutics. Amyloid. 2018;25(3):148\u201355.","journal-title":"Amyloid"},{"key":"4464_CR48","doi-asserted-by":"publisher","first-page":"103215","DOI":"10.1016\/j.scr.2023.103215","volume":"72","author":"B Bonilauri","year":"2023","unstructured":"Bonilauri B, Shin HS, Htet M, Yan CD, Witteles RM, Sallam K, et al. Generation of two induced pluripotent stem cell lines from patients with cardiac amyloidosis carrying heterozygous transthyretin (TTR) mutation. Stem Cell Res. 2023;72:103215.","journal-title":"Stem Cell Res"},{"issue":"2","key":"4464_CR49","doi-asserted-by":"publisher","first-page":"e24590","DOI":"10.1016\/j.heliyon.2024.e24590","volume":"10","author":"K Ouchi","year":"2024","unstructured":"Ouchi K, Isono K, Ohya Y, Shiraki N, Tasaki M, Inomata Y, et al. Characterization of heterozygous ATTR Tyr114Cys amyloidosis-specific induced pluripotent stem cells. Heliyon. 2024;10(2):e24590.","journal-title":"Heliyon"},{"issue":"26","key":"4464_CR50","doi-asserted-by":"publisher","first-page":"14943","DOI":"10.1073\/pnas.261419998","volume":"98","author":"X Jiang","year":"2001","unstructured":"Jiang X, Buxbaum JN, Kelly JW. The V122I cardiomyopathy variant of transthyretin increases the velocity of rate-limiting tetramer dissociation, resulting in accelerated amyloidosis. Proc Natl Acad Sci USA. 2001;98(26):14943\u20138.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"1","key":"4464_CR51","doi-asserted-by":"publisher","first-page":"581","DOI":"10.1038\/s41467-024-44820-3","volume":"15","author":"BA Nguyen","year":"2024","unstructured":"Nguyen BA, Singh V, Afrin S, Yakubovska A, Wang L, Ahmed Y, et al. Structural polymorphism of amyloid fibrils in ATTR amyloidosis revealed by cryo-electron microscopy. Nat Commun. 2024;15(1):581.","journal-title":"Nat Commun"},{"issue":"29","key":"4464_CR52","doi-asserted-by":"publisher","first-page":"E6741","DOI":"10.1073\/pnas.1805131115","volume":"115","author":"L Saelices","year":"2018","unstructured":"Saelices L, Chung K, Lee JH, Cohn W, Whitelegge JP, Benson MD, et al. Amyloid seeding of transthyretin by ex vivo cardiac fibrils and its inhibition. Proc Natl Acad Sci USA. 2018;115(29):E6741\u201350.","journal-title":"Proc Natl Acad Sci USA"},{"issue":"3","key":"4464_CR53","doi-asserted-by":"publisher","first-page":"598","DOI":"10.1002\/ejhf.3137","volume":"26","author":"L Chacko","year":"2024","unstructured":"Chacko L, Kotecha T, Ioannou A, Patel N, Martinez-Naharro A, Razvi Y, et al. Myocardial perfusion in cardiac amyloidosis. Eur J Heart Fail. 2024;26(3):598\u2013609.","journal-title":"Eur J Heart Fail"},{"issue":"5","key":"4464_CR54","doi-asserted-by":"publisher","first-page":"648","DOI":"10.1111\/his.12793","volume":"68","author":"BT Larsen","year":"2016","unstructured":"Larsen BT, Mereuta OM, Dasari S, Fayyaz AU, Theis JD, Vrana JA, et al. Correlation of histomorphological pattern of cardiac amyloid deposition with amyloid type: a histological and proteomic analysis of 108 cases. Histopathology. 2016;68(5):648\u201356.","journal-title":"Histopathology"},{"key":"4464_CR55","doi-asserted-by":"crossref","unstructured":"Alencar Neto AC, Cafezeiro CRF, Bueno BVK, Ribeiro De Souza F, Henrique Rissato JHS, Souza Borges T et al. Coronary flow reserve by PET 13\u00a0N-ammonia in patients with hereditary transthyretin amyloidosis with and without cardiac involvement. Eur Heart J. 2022;43(Supplement_2).","DOI":"10.1093\/eurheartj\/ehac544.1778"},{"key":"4464_CR56","doi-asserted-by":"publisher","first-page":"89","DOI":"10.1016\/j.yjmcc.2020.08.018","volume":"148","author":"TG Martin","year":"2020","unstructured":"Martin TG, Kirk JA. Under construction: the dynamic assembly, maintenance, and degradation of the cardiac sarcomere. J Mol Cell Cardiol. 2020;148:89\u2013102.","journal-title":"J Mol Cell Cardiol"},{"issue":"44","key":"4464_CR57","doi-asserted-by":"publisher","first-page":"31752","DOI":"10.1074\/jbc.M113.469858","volume":"288","author":"RJ Nunes","year":"2013","unstructured":"Nunes RJ, de Oliveira P, Lages A, Becker JD, Marcelino P, Barroso E, et al. Transthyretin proteins regulate angiogenesis by conferring different molecular identities to endothelial cells. J Biol Chem. 2013;288(44):31752\u201360.","journal-title":"J Biol Chem"},{"issue":"8","key":"4464_CR58","doi-asserted-by":"publisher","first-page":"911","DOI":"10.1038\/labinvest.2013.83","volume":"93","author":"Y Misumi","year":"2013","unstructured":"Misumi Y, Ando Y, Gon\u00e7alves NP, Saraiva MJ. Fibroblasts endocytose and degrade transthyretin aggregates in transthyretin-related amyloidosis. Lab Invest. 2013;93(8):911\u201320.","journal-title":"Lab Invest"}],"container-title":["Stem Cell Research & Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13287-025-04464-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1186\/s13287-025-04464-6\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1186\/s13287-025-04464-6.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,7,6]],"date-time":"2025-07-06T15:02:18Z","timestamp":1751814138000},"score":1,"resource":{"primary":{"URL":"https:\/\/stemcellres.biomedcentral.com\/articles\/10.1186\/s13287-025-04464-6"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,7,6]]},"references-count":58,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2025,12]]}},"alternative-id":["4464"],"URL":"https:\/\/doi.org\/10.1186\/s13287-025-04464-6","relation":{},"ISSN":["1757-6512"],"issn-type":[{"value":"1757-6512","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,7,6]]},"assertion":[{"value":"25 October 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"13 June 2025","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"6 July 2025","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"The cardiomyocytes and fibroblasts are obtained from two iPSC lines (UKKi032-C and UKKi037-C), which are provided under an MTA with the Uniklinik Koln. There the original material was obtained from material of healthy donors under ethical approval. All subjects provided signed informed consent prior to participation on 14-7-2016 and 25-8-2016. The IPSC lines are deposited at the European Bank for induced Pluripotent Stem Cells (EBiSC, ) and listed at the international online registry hPSCreg (). The use of human iPSCs for research purposes is approved by the ethics committee (TCBio, title: UNRAVEL, approval number: 12\u2013387, 19\u20139-2023) of University Medical Center Utrecht (Utrecht, the Netherlands). Lonza has confirmed that there was initial ethical approval for collection of HUVECs, and that the donors had signed informed consent. All experiments were conducted according to the criteria of the code of proper use of human tissue used in the Netherlands.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethics approval and consent to participate"}},{"value":"Not applicable.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Consent for publication"}},{"value":"The UMCU, which employs MO, received consultancy fees from Pfizer, Alnylam, AstraZeneca, Novartis, and Janssen Cilag. MM declares hospitality from Janssen Cilag, Speakers bureau: Janssen Cilag, Pfizer, Siemens, Springer Healthcare, Consultancy: Janssen Cilag, CDR Life, GSK, BMS, all paid to institution. The other authors report no competing interests and no relationships relevant to this paper\u2019s contents to disclose.","order":4,"name":"Ethics","group":{"name":"EthicsHeading","label":"Competing interests"}},{"value":"The authors declare that they have not use AI-generated work in this manuscript.","order":5,"name":"Ethics","group":{"name":"EthicsHeading","label":"Use of AI"}}],"article-number":"352"}}