{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,20]],"date-time":"2026-08-20T15:45:19Z","timestamp":1787240719215,"version":"build-2736575974"},"reference-count":75,"publisher":"MDPI AG","issue":"3","license":[{"start":{"date-parts":[[2026,3,18]],"date-time":"2026-03-18T00:00:00Z","timestamp":1773792000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Genes"],"abstract":"<jats:p>Transthyretin amyloidosis (ATTR) is a progressive disease characterized by systemic deposition of transthyretin-derived amyloid. Although the recent advent of disease-modifying therapies has expanded treatment options, substantial unmet needs remain, such as understanding disease heterogeneity, predicting treatment response, and prognostic stratification. In this review, we highlight the current and emerging roles of omics technologies in both clinical and research settings of ATTR, including genomics and its integration with other modalities. Currently, omics technologies are applied in clinical settings for accurate disease typing. Clinical samples are utilized to identify risk factors beyond specific transthyretin variants via genomics and epigenomics and to discover promising biomarkers via proteomics. Accumulating findings from omics analyses using cell and animal models are also facilitating the elucidation of the complex pathology of ATTR. Nevertheless, the application of omics analysis in ATTR research is still developing. Moving forward, it is expected to play a central role in accumulating datasets, leveraging cutting-edge technologies, utilizing integrated multi-omics, and bridging basic and clinical research. These advancements are expected to further accelerate the implementation of next-generation therapeutic strategies and precision medicine.<\/jats:p>","DOI":"10.3390\/genes17030333","type":"journal-article","created":{"date-parts":[[2026,3,18]],"date-time":"2026-03-18T11:14:14Z","timestamp":1773832454000},"page":"333","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Application of Omics Analysis in the Clinical Practice and Research of Transthyretin Amyloidosis"],"prefix":"10.3390","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-8045-113X","authenticated-orcid":false,"given":"Hidenori","family":"Moriyama","sequence":"first","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Faiyza Akil","family":"Shaikh","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7316-3546","authenticated-orcid":false,"given":"Toshifumi","family":"Yokota","sequence":"additional","affiliation":[{"name":"Department of Medical Genetics, Faculty of Medicine and Dentistry, University of Alberta, Edmonton, AB T6G 2H7, Canada"},{"name":"The Friends of Garrett Cumming Research & Muscular Dystrophy Canada, HM Toupin Neurological Sciences Research, Edmonton, AB T6G 2H7, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2026,3,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2872","DOI":"10.1016\/j.jacc.2019.04.003","article-title":"Transthyretin Amyloid Cardiomyopathy: JACC State-of-the-Art Review","volume":"73","author":"Ruberg","year":"2019","journal-title":"J. Am. Coll. Cardiol."},{"key":"ref_2","doi-asserted-by":"crossref","first-page":"1610","DOI":"10.1253\/circj.CJ-20-0110","article-title":"JCS 2020 Guideline on Diagnosis and Treatment of Cardiac Amyloidosis","volume":"84","author":"Kitaoka","year":"2020","journal-title":"Circ. J."},{"key":"ref_3","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1111\/joim.13169","article-title":"Systemic amyloidosis from A (AA) to T (ATTR): A review","volume":"289","author":"Muchtar","year":"2021","journal-title":"J. Intern. Med."},{"key":"ref_4","doi-asserted-by":"crossref","first-page":"15525","DOI":"10.1021\/bi051608t","article-title":"The pathway by which the tetrameric protein transthyretin dissociates","volume":"44","author":"Foss","year":"2005","journal-title":"Biochemistry"},{"key":"ref_5","doi-asserted-by":"crossref","first-page":"6470","DOI":"10.1021\/bi952501g","article-title":"The acid-mediated denaturation pathway of transthyretin yields a conformational intermediate that can self-assemble into amyloid","volume":"35","author":"Lai","year":"1996","journal-title":"Biochemistry"},{"key":"ref_6","doi-asserted-by":"crossref","first-page":"27207","DOI":"10.1074\/jbc.M101024200","article-title":"Tetramer dissociation and monomer partial unfolding precedes protofibril formation in amyloidogenic transthyretin variants","volume":"276","author":"Quintas","year":"2001","journal-title":"J. Biol. Chem."},{"key":"ref_7","doi-asserted-by":"crossref","first-page":"999","DOI":"10.1093\/eurheartj\/ehae811","article-title":"Transthyretin amyloid cardiomyopathy: A paradigm for advancing precision medicine","volume":"46","author":"Maurer","year":"2025","journal-title":"Eur. Heart J."},{"key":"ref_8","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1038\/nrcardio.2014.165","article-title":"Pathophysiology and treatment of cardiac amyloidosis","volume":"12","author":"Gertz","year":"2015","journal-title":"Nat. Rev. Cardiol."},{"key":"ref_9","doi-asserted-by":"crossref","first-page":"35028","DOI":"10.1038\/s41598-025-21745-5","article-title":"Polyneuropathy in hereditary and wildtype transthyretin amyloidosis, comparison of key clinical features and red flags","volume":"15","author":"Siemer","year":"2025","journal-title":"Sci. Rep."},{"key":"ref_10","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1016\/j.humpath.2010.11.017","article-title":"Wild-type transthyretin-derived amyloidosis in various ligaments and tendons","volume":"42","author":"Sueyoshi","year":"2011","journal-title":"Hum. Pathol."},{"key":"ref_11","doi-asserted-by":"crossref","first-page":"778","DOI":"10.1001\/jama.2024.0442","article-title":"Cardiac Amyloidosis Due to Transthyretin Protein: A Review","volume":"331","author":"Ruberg","year":"2024","journal-title":"JAMA"},{"key":"ref_12","doi-asserted-by":"crossref","first-page":"58","DOI":"10.3109\/13506129.2015.1135792","article-title":"Carpal tunnel syndrome: A common initial symptom of systemic wild-type ATTR (ATTRwt) amyloidosis","volume":"23","author":"Nakagawa","year":"2016","journal-title":"Amyloid"},{"key":"ref_13","doi-asserted-by":"crossref","first-page":"2585","DOI":"10.1093\/eurheartj\/ehv338","article-title":"Wild-type transthyretin amyloidosis as a cause of heart failure with preserved ejection fraction","volume":"36","author":"Robles","year":"2015","journal-title":"Eur. Heart J."},{"key":"ref_14","doi-asserted-by":"crossref","first-page":"2879","DOI":"10.1093\/eurheartj\/ehx350","article-title":"Unveiling transthyretin cardiac amyloidosis and its predictors among elderly patients with severe aortic stenosis undergoing transcatheter aortic valve replacement","volume":"38","author":"Narotsky","year":"2017","journal-title":"Eur. Heart J."},{"key":"ref_15","doi-asserted-by":"crossref","first-page":"1267","DOI":"10.1001\/jamacardio.2021.3070","article-title":"Prevalence of Transthyretin Amyloid Cardiomyopathy in Heart Failure With Preserved Ejection Fraction","volume":"6","author":"AbouEzzeddine","year":"2021","journal-title":"JAMA Cardiol."},{"key":"ref_16","doi-asserted-by":"crossref","unstructured":"Poli, L., Labella, B., Cotti Piccinelli, S., Caria, F., Risi, B., Damioli, S., Padovani, A., and Filosto, M. (2023). Hereditary transthyretin amyloidosis: A comprehensive review with a focus on peripheral neuropathy. Front. Neurol., 14.","DOI":"10.3389\/fneur.2023.1242815"},{"key":"ref_17","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1038\/s41582-019-0210-4","article-title":"Hereditary transthyretin amyloidosis: A model of medical progress for a fatal disease","volume":"15","author":"Adams","year":"2019","journal-title":"Nat. Rev. Neurol."},{"key":"ref_18","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1016\/j.jacc.2025.09.004","article-title":"Transthyretin Cardiac Amyloidosis Evaluation and Management: 2025 ACC Concise Clinical Guidance","volume":"87","author":"Kittleson","year":"2026","journal-title":"J. Am. Coll. Cardiol."},{"key":"ref_19","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1136\/heartjnl-2024-325184","article-title":"Current and emerging treatment options for transthyretin amyloid cardiomyopathy","volume":"112","author":"Vergaro","year":"2026","journal-title":"Heart"},{"key":"ref_20","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1097\/HCO.0000000000001198","article-title":"New therapies to treat cardiac amyloidosis","volume":"40","author":"Nguyen","year":"2025","journal-title":"Curr. Opin. Cardiol."},{"key":"ref_21","doi-asserted-by":"crossref","first-page":"e008193","DOI":"10.1161\/CIRCHEARTFAILURE.120.008193","article-title":"Long-Term Survival With Tafamidis in Patients With Transthyretin Amyloid Cardiomyopathy","volume":"15","author":"Elliott","year":"2022","journal-title":"Circ. Heart Fail."},{"key":"ref_22","doi-asserted-by":"crossref","first-page":"132","DOI":"10.1056\/NEJMoa2305434","article-title":"Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy","volume":"390","author":"Gillmore","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"ref_23","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1056\/NEJMoa2409134","article-title":"Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy","volume":"392","author":"Fontana","year":"2025","journal-title":"N. Engl. J. Med."},{"key":"ref_24","doi-asserted-by":"crossref","first-page":"493","DOI":"10.1056\/NEJMoa2107454","article-title":"CRISPR-Cas9 In Vivo Gene Editing for Transthyretin Amyloidosis","volume":"385","author":"Gillmore","year":"2021","journal-title":"N. Engl. J. Med."},{"key":"ref_25","doi-asserted-by":"crossref","first-page":"2231","DOI":"10.1056\/NEJMoa2412309","article-title":"CRISPR-Cas9 Gene Editing with Nexiguran Ziclumeran for ATTR Cardiomyopathy","volume":"391","author":"Fontana","year":"2024","journal-title":"N. Engl. J. Med."},{"key":"ref_26","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1056\/NEJMoa2303765","article-title":"Phase 1 Trial of Antibody NI006 for Depletion of Cardiac Transthyretin Amyloid","volume":"389","author":"Donal","year":"2023","journal-title":"N. Engl. J. Med."},{"key":"ref_27","doi-asserted-by":"crossref","first-page":"e012112","DOI":"10.1161\/CIRCHEARTFAILURE.124.012112","article-title":"Changing Treatment Landscape in Transthyretin Cardiac Amyloidosis","volume":"18","author":"Fontana","year":"2025","journal-title":"Circ. Heart Fail."},{"key":"ref_28","doi-asserted-by":"crossref","unstructured":"Hayes, C.N., Nakahara, H., Ono, A., Tsuge, M., and Oka, S. (2024). From Omics to Multi-Omics: A Review of Advantages and Tradeoffs. Genes, 15.","DOI":"10.20944\/preprints202411.0882.v1"},{"key":"ref_29","doi-asserted-by":"crossref","unstructured":"Dai, X., and Shen, L. (2022). Advances and Trends in Omics Technology Development. Front. Med., 9.","DOI":"10.3389\/fmed.2022.911861"},{"key":"ref_30","doi-asserted-by":"crossref","unstructured":"Campanelli, L., Sendoya, J.M., Brody, S., Galeano, P., Do Carmo, S., Cuello, A.C., Casta\u00f1o, E.M., Gonzal\u00e9s-Jimenez, A., Verheul, J., and Medina-Vera, D. (2025). New insights into the molecular biology of Alzheimer\u2019s-like cerebral amyloidosis achieved through multi-omics approaches. PLoS ONE, 20.","DOI":"10.1371\/journal.pone.0336509"},{"key":"ref_31","doi-asserted-by":"crossref","first-page":"9","DOI":"10.1016\/j.clinbiochem.2018.06.011","article-title":"Alzheimer\u2019s disease in the omics era","volume":"59","author":"Sancesario","year":"2018","journal-title":"Clin. Biochem."},{"key":"ref_32","doi-asserted-by":"crossref","first-page":"412","DOI":"10.1038\/s41588-022-01024-z","article-title":"New insights into the genetic etiology of Alzheimer\u2019s disease and related dementias","volume":"54","author":"Bellenguez","year":"2022","journal-title":"Nat. Genet."},{"key":"ref_33","doi-asserted-by":"crossref","first-page":"1852","DOI":"10.1016\/j.mayocp.2020.06.029","article-title":"Amyloid Typing by Mass Spectrometry in Clinical Practice: A Comprehensive Review of 16,175 Samples","volume":"95","author":"Dasari","year":"2020","journal-title":"Mayo Clin. Proc."},{"key":"ref_34","doi-asserted-by":"crossref","first-page":"4957","DOI":"10.1182\/blood-2009-07-230722","article-title":"Classification of amyloidosis by laser microdissection and mass spectrometry-based proteomic analysis in clinical biopsy specimens","volume":"114","author":"Vrana","year":"2009","journal-title":"Blood"},{"key":"ref_35","doi-asserted-by":"crossref","first-page":"1239","DOI":"10.3324\/haematol.2013.102764","article-title":"Clinical diagnosis and typing of systemic amyloidosis in subcutaneous fat aspirates by mass spectrometry-based proteomics","volume":"99","author":"Vrana","year":"2014","journal-title":"Haematologica"},{"key":"ref_36","doi-asserted-by":"crossref","first-page":"226","DOI":"10.1038\/ki.2012.108","article-title":"Laser microdissection and mass spectrometry-based proteomics aids the diagnosis and typing of renal amyloidosis","volume":"82","author":"Sethi","year":"2012","journal-title":"Kidney Int."},{"key":"ref_37","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1002\/cjp2.126","article-title":"The complementary role of histology and proteomics for diagnosis and typing of systemic amyloidosis","volume":"5","author":"Rezk","year":"2019","journal-title":"J. Pathol. Clin. Res."},{"key":"ref_38","doi-asserted-by":"crossref","first-page":"1474","DOI":"10.1002\/ehf2.13773","article-title":"Step-by-step typing for the accurate diagnosis of concurrent light chain and transthyretin cardiac amyloidosis","volume":"9","author":"Moriyama","year":"2022","journal-title":"ESC Heart Fail."},{"key":"ref_39","doi-asserted-by":"crossref","first-page":"4104","DOI":"10.1021\/acs.jproteome.7b00479","article-title":"Serum Proteomic Variability Associated with Clinical Phenotype in Familial Transthyretin Amyloidosis (ATTRm)","volume":"16","author":"Chan","year":"2017","journal-title":"J. Proteome Res."},{"key":"ref_40","doi-asserted-by":"crossref","first-page":"1659","DOI":"10.1021\/acs.jproteome.6b00998","article-title":"Blood Proteomic Profiling in Inherited (ATTRm) and Acquired (ATTRwt) Forms of Transthyretin-Associated Cardiac Amyloidosis","volume":"16","author":"Chan","year":"2017","journal-title":"J. Proteome Res."},{"key":"ref_41","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1056\/NEJMoa1716153","article-title":"Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis","volume":"379","author":"Adams","year":"2018","journal-title":"N. Engl. J. Med."},{"key":"ref_42","doi-asserted-by":"crossref","first-page":"e412","DOI":"10.1212\/WNL.0000000000011090","article-title":"Neurofilament Light Chain as a Biomarker of Hereditary Transthyretin-Mediated Amyloidosis","volume":"96","author":"Ticau","year":"2021","journal-title":"Neurology"},{"key":"ref_43","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1080\/13506129.2024.2313218","article-title":"Serum neurofilament light chain in hereditary transthyretin amyloidosis: Validation in real-life practice","volume":"31","author":"Carroll","year":"2024","journal-title":"Amyloid"},{"key":"ref_44","doi-asserted-by":"crossref","unstructured":"Nugroho, K., Lin, C.Y., Monteiro, C., Coelho, T., Moresco, J.J., Pinto, A.F.M., Powers, E.T., Yates, J.R., Diedrich, J.K., and Kelly, J.W. (2025). Plasma Proteome Profiling Reveals Inflammation Markers and Tafamidis Effects in V30M Transthyretin Polyneuropathy. Int. J. Mol. Sci., 26.","DOI":"10.3390\/ijms26125534"},{"key":"ref_45","doi-asserted-by":"crossref","first-page":"234","DOI":"10.1080\/13506129.2021.1958775","article-title":"Metabolomics analysis for diagnosis and biomarker discovery of transthyretin amyloidosis","volume":"28","author":"Olsson","year":"2021","journal-title":"Amyloid"},{"key":"ref_46","doi-asserted-by":"crossref","unstructured":"Luigetti, M., Guglielmino, V., Romano, A., Sciarrone, M.A., Vitali, F., Sabino, A., Gervasoni, J., Primiano, A., Santucci, L., and Moroni, R. (2022). A Metabolic Signature of Hereditary Transthyretin Amyloidosis: A Pilot Study. Int. J. Mol. Sci., 23.","DOI":"10.3390\/ijms232416133"},{"key":"ref_47","doi-asserted-by":"crossref","unstructured":"Wa\u015b, J., Gawor-Prokopczyk, M., Sioma, A., Szewczyk, R., Pel, A., Krzyszto\u0144-Russjan, J., Niedolistek, M., Soko\u0142owska, D., Grzybowski, J., and Mazurkiewicz, \u0141. (2025). Proteomic Analysis of Serum in Cardiac Transthyretin Amyloidosis: Diagnostic and Prognostic Implications for Biomarker Discovery. Biomedicines, 13.","DOI":"10.3390\/biomedicines13071647"},{"key":"ref_48","doi-asserted-by":"crossref","first-page":"e012434","DOI":"10.1161\/CIRCHEARTFAILURE.124.012434","article-title":"Comprehensive Proteomics Profiling Identifies Circulating Biomarkers to Distinguish Hypertrophic Cardiomyopathy From Other Cardiomyopathies With Left Ventricular Hypertrophy","volume":"18","author":"Akita","year":"2025","journal-title":"Circ. Heart Fail."},{"key":"ref_49","doi-asserted-by":"crossref","first-page":"632","DOI":"10.1016\/j.jaccao.2020.08.013","article-title":"A Proteomic Atlas of Cardiac Amyloid Plaques","volume":"2","author":"Kourelis","year":"2020","journal-title":"JACC CardioOncol."},{"key":"ref_50","doi-asserted-by":"crossref","first-page":"915","DOI":"10.2215\/CJN.07030712","article-title":"Laser microdissection and proteomic analysis of amyloidosis, cryoglobulinemic GN, fibrillary GN, and immunotactoid glomerulopathy","volume":"8","author":"Sethi","year":"2013","journal-title":"Clin. J. Am. Soc. Nephrol."},{"key":"ref_51","doi-asserted-by":"crossref","first-page":"72","DOI":"10.1080\/13506129.2024.2448440","article-title":"Whole tissue proteomic analyses of cardiac ATTR and AL unveil mechanisms of tissue damage","volume":"32","author":"Netzel","year":"2025","journal-title":"Amyloid"},{"key":"ref_52","doi-asserted-by":"crossref","first-page":"550","DOI":"10.1016\/j.jaccao.2021.07.002","article-title":"Genomic Screening for Pathogenic Transthyretin Variants Finds Evidence of Underdiagnosed Amyloid Cardiomyopathy From Health Records","volume":"3","author":"Carry","year":"2021","journal-title":"JACC CardioOncol."},{"key":"ref_53","doi-asserted-by":"crossref","first-page":"964","DOI":"10.1001\/jamacardio.2024.2190","article-title":"Prevalence, Cardiac Phenotype, and Outcomes of Transthyretin Variants in the UK Biobank Population","volume":"9","author":"Aung","year":"2024","journal-title":"JAMA Cardiol."},{"key":"ref_54","doi-asserted-by":"crossref","first-page":"ytae199","DOI":"10.1093\/ehjcr\/ytae199","article-title":"Familial occurrences of cardiac wild-type transthyretin amyloidosis: A case series","volume":"8","author":"Westin","year":"2024","journal-title":"Eur. Heart J. Case Rep."},{"key":"ref_55","doi-asserted-by":"crossref","unstructured":"Moreno-G\u00e1zquez, I., P\u00e9rez-Palacios, R., Abengochea-Qu\u00edlez, L., Lahuerta Pueyo, C., Roteta Unceta Barrenechea, A., Andr\u00e9s Gracia, A., Aibar Arregui, M.A., and Menao Guill\u00e9n, S. (2023). Targeted sequencing of selected functional genes in patients with wild-type transthyretin amyloidosis. BMC Res. Notes, 16.","DOI":"10.1186\/s13104-023-06491-z"},{"key":"ref_56","unstructured":"(2026, January 25). TTR GWAS. Available online: https:\/\/ttrgwas.wordpress.com\/?utm_source=chatgpt.com."},{"key":"ref_57","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1111\/joim.70020","article-title":"Sixty years of experience with hereditary transthyretin amyloidosis: Insights from the Swedish transthyretin amyloidosis registry","volume":"298","author":"Stenberg","year":"2025","journal-title":"J. Intern. Med."},{"key":"ref_58","doi-asserted-by":"crossref","first-page":"S3","DOI":"10.1097\/WCO.0000000000000288","article-title":"Sixty years of transthyretin familial amyloid polyneuropathy (TTR-FAP) in Europe: Where are we now? A European network approach to defining the epidemiology and management patterns for TTR-FAP","volume":"29","author":"Parman","year":"2016","journal-title":"Curr. Opin. Neurol."},{"key":"ref_59","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1186\/s13148-020-00967-6","article-title":"Epigenetic profiling of Italian patients identified methylation sites associated with hereditary transthyretin amyloidosis","volume":"12","author":"Pathak","year":"2020","journal-title":"Clin. Epigenetics"},{"key":"ref_60","doi-asserted-by":"crossref","first-page":"e003011","DOI":"10.1161\/CIRCGEN.120.003011","article-title":"Epigenomic Profiles of African-American Transthyretin Val122Ile Carriers Reveals Putatively Dysregulated Amyloid Mechanisms","volume":"14","author":"Pathak","year":"2021","journal-title":"Circ. Genom. Precis. Med."},{"key":"ref_61","doi-asserted-by":"crossref","first-page":"H149","DOI":"10.1152\/ajpheart.00073.2021","article-title":"Transthyretin amyloid fibrils alter primary fibroblast structure, function, and inflammatory gene expression","volume":"321","author":"Dittloff","year":"2021","journal-title":"Am. J. Physiol. Heart Circ. Physiol."},{"key":"ref_62","doi-asserted-by":"crossref","unstructured":"Azevedo, E.P., Guimaraes-Costa, A.B., Bandeira-Melo, C., Chimelli, L., Waddington-Cruz, M., Saraiva, E.M., Palhano, F.L., and Foguel, D. (2019). Inflammatory profiling of patients with familial amyloid polyneuropathy. BMC Neurol., 19.","DOI":"10.1186\/s12883-019-1369-4"},{"key":"ref_63","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1186\/s13287-025-04464-6","article-title":"Uncovering cell type-specific phenotypes using a novel human in vitro model of transthyretin amyloid cardiomyopathy","volume":"16","author":"Qin","year":"2025","journal-title":"Stem Cell Res. Ther."},{"key":"ref_64","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1080\/13506129.2023.2224494","article-title":"Mapping cellular response to destabilized transthyretin reveals cell- and amyloidogenic protein-specific signatures","volume":"30","author":"Ghosh","year":"2023","journal-title":"Amyloid"},{"key":"ref_65","doi-asserted-by":"crossref","first-page":"2817","DOI":"10.1073\/pnas.0400062101","article-title":"Tissue damage in the amyloidoses: Transthyretin monomers and nonnative oligomers are the major cytotoxic species in tissue culture","volume":"101","author":"Reixach","year":"2004","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"ref_66","doi-asserted-by":"crossref","first-page":"451","DOI":"10.1016\/j.stemcr.2013.10.003","article-title":"Induced pluripotent stem cell modeling of multisystemic, hereditary transthyretin amyloidosis","volume":"1","author":"Leung","year":"2013","journal-title":"Stem Cell Rep."},{"key":"ref_67","doi-asserted-by":"crossref","first-page":"515","DOI":"10.1016\/j.stemcr.2020.07.003","article-title":"Expression of Amyloidogenic Transthyretin Drives Hepatic Proteostasis Remodeling in an Induced Pluripotent Stem Cell Model of Systemic Amyloid Disease","volume":"15","author":"Giadone","year":"2020","journal-title":"Stem Cell Rep."},{"key":"ref_68","doi-asserted-by":"crossref","unstructured":"Ibrahim, R.B., Liu, Y.T., Yeh, S.Y., and Tsai, J.W. (2019). Contributions of Animal Models to the Mechanisms and Therapies of Transthyretin Amyloidosis. Front. Physiol., 10.","DOI":"10.3389\/fphys.2019.00338"},{"key":"ref_69","doi-asserted-by":"crossref","first-page":"1997","DOI":"10.1093\/cvr\/cvaf152","article-title":"In vitro and in vivo disease models of cardiac amyloidosis: Progress, pitfalls, and potential","volume":"121","author":"Qin","year":"2025","journal-title":"Cardiovasc. Res."},{"key":"ref_70","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/j.bbrc.2022.02.035","article-title":"TTR exon-humanized mouse optimal for verifying new therapies for FAP","volume":"599","author":"Li","year":"2022","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"ref_71","doi-asserted-by":"crossref","unstructured":"Ishida, S., Sato, Y., Chosa, K., Ezawa, E., Yamauchi, Y., Oyama, M., Kozuka-Hata, H., Ito, R., Sato, R., and Maeki, M. (2026). CRISPR-Cas3-based editing for targeted deletions in a mouse model of transthyretin amyloidosis. Nat. Biotechnol.","DOI":"10.1038\/s41587-025-02949-6"},{"key":"ref_72","doi-asserted-by":"crossref","first-page":"2893","DOI":"10.1093\/eurheartj\/ehad347","article-title":"Conventional heart failure therapy in cardiac ATTR amyloidosis","volume":"44","author":"Ioannou","year":"2023","journal-title":"Eur. Heart J."},{"key":"ref_73","doi-asserted-by":"crossref","first-page":"e022859","DOI":"10.1161\/JAHA.121.022859","article-title":"Lack of Association Between Neurohormonal Blockade and Survival in Transthyretin Cardiac Amyloidosis","volume":"10","author":"Cheng","year":"2021","journal-title":"J. Am. Heart Assoc."},{"key":"ref_74","doi-asserted-by":"crossref","unstructured":"Clark, C., Rabl, M., Dayon, L., and Popp, J. (2022). The promise of multi-omics approaches to discover biological alterations with clinical relevance in Alzheimer\u2019s disease. Front. Aging Neurosci., 14.","DOI":"10.3389\/fnagi.2022.1065904"},{"key":"ref_75","doi-asserted-by":"crossref","first-page":"3288","DOI":"10.1016\/j.ymthe.2024.08.030","article-title":"Integrative proteomic and metabolomic elucidation of cardiomyopathy with in vivo and in vitro models and clinical samples","volume":"32","author":"Hu","year":"2024","journal-title":"Mol. Ther."}],"container-title":["Genes"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.mdpi.com\/2073-4425\/17\/3\/333\/pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,3,20]],"date-time":"2026-03-20T05:23:54Z","timestamp":1773984234000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.mdpi.com\/2073-4425\/17\/3\/333"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,3,18]]},"references-count":75,"journal-issue":{"issue":"3","published-online":{"date-parts":[[2026,3]]}},"alternative-id":["genes17030333"],"URL":"https:\/\/doi.org\/10.3390\/genes17030333","relation":{},"ISSN":["2073-4425"],"issn-type":[{"value":"2073-4425","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,3,18]]}}}