Döhner H, Wei AH, Appelbaum FR, Craddock C, DiNardo CD, Dombret H, et al. Diagnosis and management of AML in adults: 2022 recommendations from an international expert panel on behalf of the ELN. Blood. 2022;140:1345–77.
Kantarjian H, Borthakur G, Daver N, DiNardo CD, Issa G, Jabbour E, et al. Current status and research directions in acute myeloid leukemia. Blood Cancer J. 2024;14:163.
Trabolsi A, Arumov A, Schatz JH. Bispecific antibodies and CAR-T cells: dueling immunotherapies for large B-cell lymphomas. Blood Cancer J. 2024;14:27.
Haubner S, Subklewe M, Sadelain M. Honing CAR T cells to tackle acute myeloid leukemia. Blood. 2025;145:1113–25.
D’Angelo SP, Araujo DM, Abdul Razak AR, Agulnik M, Attia S, Blay JY, et al. Afamitresgene autoleucel for advanced synovial sarcoma and myxoid round cell liposarcoma (SPEARHEAD-1): an international, open-label, phase 2 trial. Lancet. 2024;403:1460–71.
Kim SP, Vale NR, Zacharakis N, Krishna S, Yu Z, Gasmi B, et al. Adoptive cellular therapy with autologous tumor-infiltrating lymphocytes and T-cell receptor-engineered T cells targeting common p53 neoantigens in human solid tumors. Cancer Immunol Res. 2022;10:932–46.
Leidner R, Sanjuan Silva N, Huang H, Sprott D, Zheng C, Shih YP, et al. Neoantigen T-cell receptor gene therapy in pancreatic cancer. N Engl J Med. 2022;386:2112–9.
van Amerongen RA, Hagedoorn RS, Remst DFG, Assendelft DC, van der Steen DM, Wouters AK, et al. WT1-specific TCRs directed against newly identified peptides install antitumor reactivity against acute myeloid leukemia and ovarian carcinoma. J Immunotherapy Cancer 2022;10:e004409.
Amir AL, van der Steen DM, van Loenen MM, Hagedoorn RS, de Boer R, Kester MD, et al. PRAME-specific Allo-HLA-restricted T cells with potent antitumor reactivity useful for therapeutic T-cell receptor gene transfer. Clin Cancer Res. 2011;17:5615–25.
Li Y, Moysey R, Molloy PE, Vuidepot AL, Mahon T, Baston E, et al. Directed evolution of human T-cell receptors with picomolar affinities by phage display. Nat Biotechnol. 2005;23:349–54.
Foldvari Z, Brennan MS, Titov A, Jacobsen SEW, Olweus J Targeting the roots of myeloid malignancies with T cell receptors. Nat Rev Cancer. 2025. [Epub ahead of print]. https://doi.org/10.1038/s41568-025-00857-0.
Gore S, Blyth E, Bleakley M, Lee K, Micklethwaite K, Gowrishankar K. Current developments in T-cell receptor therapy for acute myeloid leukemia. Blood Adv. 2025;9:3069–89.
van der Lee DI, Reijmers RM, Honders MW, Hagedoorn RS, de Jong RC, Kester MG, et al. Mutated nucleophosmin 1 as immunotherapy target in acute myeloid leukemia. J Clin Investig. 2019;129:774–85.
van der Lee DI, Koutsoumpli G, Reijmers RM, Honders MW, de Jong RCM, Remst DFG, et al. An HLA-A*11:01-binding neoantigen from mutated NPM1 as target for TCR gene therapy in AML. Cancers (Basel). 2021;13:5390.
Giannakopoulou E, Lehander M, Virding Culleton S, Yang W, Li Y, Karpanen T, et al. A T cell receptor targeting a recurrent driver mutation in FLT3 mediates elimination of primary human acute myeloid leukemia in vivo. Nat Cancer. 2023;4:1474–90.
Leung WK, Torres Chavez AG, French-Kim M, Shafer P, Mamonkin M, Hill LC, et al. Targeting IDH2R140Q and other neoantigens in acute myeloid leukemia. Blood. 2024;143:1726–37.
Biernacki MA, Lok J, Foster KA, Cummings C, Busch S, Black RG, et al. SF3B1K700E Neoantigen Is a CD8+ T-cell Target Shared across Human Myeloid Neoplasms. Cancer Immunol Res. 2025;13:1391–404.
Biernacki MA, Lok J, Black RG, Foster KA, Cummings C, Woodward KB, et al. Discovery of U2AF1 neoantigens in myeloid neoplasms. J Immunotherapy Cancer. 2023;11:e007490.
Biernacki MA, Foster KA, Woodward KB, Coon ME, Cummings C, Cunningham TM, et al. CBFB-MYH11 fusion neoantigen enables T cell recognition and killing of acute myeloid leukemia. J Clin Investig. 2020;130:5127–41.
Kim WJ, Crosse EI, De Neef E, Etxeberria I, Sabio EY, Wang E, et al. Mis-splicing-derived neoantigens and cognate TCRs in splicing factor mutant leukemias. Cell. 2025;188:3422–3440.e3424.
van der Werf I, Wojtuszkiewicz A, Meggendorfer M, Hutter S, Baer C, Heymans M, et al. Splicing factor gene mutations in acute myeloid leukemia offer additive value if incorporated in current risk classification. Blood Adv. 2021;5:3254–65.
Papaemmanuil E, Gerstung M, Bullinger L, Gaidzik VI, Paschka P, Roberts ND, et al. Genomic classification and prognosis in acute myeloid leukemia. N Engl J Med. 2016;374:2209–21.
Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al. The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms. Leukemia. 2022;36:1703–19.
Gaidzik VI, Teleanu V, Papaemmanuil E, Weber D, Paschka P, Hahn J, et al. RUNX1 mutations in acute myeloid leukemia are associated with distinct clinico-pathologic and genetic features. Leukemia. 2016;30:2282.
Perry C, Eldor A, Soreq H. Runx1/AML1 in leukemia: disrupted association with diverse protein partners. Leuk Res. 2002;26:221–8.
Tang JL, Hou HA, Chen CY, Liu CY, Chou WC, Tseng MH, et al. AML1/RUNX1 mutations in 470 adult patients with de novo acute myeloid leukemia: prognostic implication and interaction with other gene alterations. Blood. 2009;114:5352–61.
Harada H, Harada Y, Niimi H, Kyo T, Kimura A, Inaba T. High incidence of somatic mutations in the AML1/RUNX1 gene in myelodysplastic syndrome and low blast percentage myeloid leukemia with myelodysplasia. Blood. 2004;103:2316–24.
Gerritsen M, Yi G, Tijchon E, Kuster J, Schuringa JJ, Martens JHA, et al. RUNX1 mutations enhance self-renewal and block granulocytic differentiation in human in vitro models and primary AMLs. Blood Adv. 2019;3:320–32.
Niebuhr B, Fischer M, Täger M, Cammenga J, Stocking C. Gatekeeper function of the RUNX1 transcription factor in acute leukemia. Blood Cells Mol Dis. 2008;40:211–8.
Hu ZB, Ma W, Zaborski M, MacLeod R, Quentmeier H, Drexler HG. Establishment and characterization of two novel cytokine-responsive acute myeloid and monocytic leukemia cell lines, MUTZ-2 and MUTZ-3. Leukemia. 1996;10:1025–40.
Masterson AJ, Sombroek CC, De Gruijl TD, Graus YM, van der Vliet HJ, Lougheed SM, et al. MUTZ-3, a human cell line model for the cytokine-induced differentiation of dendritic cells from CD34+ precursors. Blood. 2002;100:701–3.
van der Lee DI, Argiro EM, Laan SNJ, Honders MW, de Jong RCM, Struckman NE, et al. Mutated DNMT3A creates a public HLADQ- binding neoantigen on acute myeloid leukemia. Front Immunol. 2025;16:1556121.
Arindrarto W, Borràs DM, de Groen RAL, van den Berg RR, Locher IJ, van Diessen S, et al. Comprehensive diagnostics of acute myeloid leukemia by whole transcriptome RNA sequencing. Leukemia. 2021;35:47–61.
Perez-Riverol Y, Bandla C, Kundu DJ, Kamatchinathan S, Bai J, Hewapathirana S, et al. The PRIDE database at 20 years: 2025 update. Nucleic acids Res. 2025;53:D543–d553.
Struckman NE, de Jong RCM, Honders MW, Smith SI, van der Lee DI, Koutsoumpli G, et al. Hotspot DNA methyltransferase 3A (DNMT3A) and isocitrate dehydrogenase 1 and 2 (IDH1/2) mutations in acute myeloid leukemia and their relevance as targets for immunotherapy. Biomedicines. 2024;12:1086.
Burrows SR, Kienzle N, Winterhalter A, Bharadwaj M, Altman JD, Brooks A. Peptide-MHC class I tetrameric complexes display exquisite ligand specificity. J Immunol. 2000;165:6229–34.
Björkström NK, Béziat V, Cichocki F, Liu LL, Levine J, Larsson S, et al. CD8 T cells express randomly selected KIRs with distinct specificities compared with NK cells. Blood. 2012;120:3455–65.
Schlott F, Steubl D, Ameres S, Moosmann A, Dreher S, Heemann U, et al. Characterization and clinical enrichment of HLA-C*07:02-restricted Cytomegalovirus-specific CD8+ T cells. PLoS One. 2018;13:e0193554.
Meeuwsen MH, Wouters AK, Jahn L, Hagedoorn RS, Kester MGD, Remst DFG, et al. A broad and systematic approach to identify B cell malignancy-targeting TCRs for multi-antigen-based T cell therapy. Mol Ther. 2022;30:564–78.
Lefranc MP. IMGT, the international ImMunoGeneTics database. Nucleic acids Res. 2001;29:207–9.
Linnemann C, Heemskerk B, Kvistborg P, Kluin RJ, Bolotin DA, Chen X, et al. High-throughput identification of antigen-specific TCRs by TCR gene capture. Nat Med. 2013;19:1534–41.
Heemskerk MH, Hoogeboom M, de Paus RA, Kester MG, van der Hoorn MA, Goulmy E, et al. Redirection of antileukemic reactivity of peripheral T lymphocytes using gene transfer of minor histocompatibility antigen HA-2-specific T-cell receptor complexes expressing a conserved alpha joining region. Blood. 2003;102:3530–40.
Matsuda M, Ono R, Iyoda T, Endo T, Iwasaki M, Tomizawa-Murasawa M, et al. Human NK cell development in hIL-7 and hIL-15 knockin NOD/SCID/IL2rgKO mice. Life Sci Alliance. 2019;2:e201800195.
Reynisson B, Alvarez B, Paul S, Peters B, Nielsen M. NetMHCpan-4.1 and NetMHCIIpan-4.0: improved predictions of MHC antigen presentation by concurrent motif deconvolution and integration of MS MHC eluted ligand data. Nucleic acids Res. 2020;48:W449–w454.
Fairley S, Lowy-Gallego E, Perry E, Flicek P. The International Genome Sample Resource (IGSR) collection of open human genomic variation resources. Nucleic acids Res. 2020;48:D941–d947.
Gonzalez-Galarza FF, McCabe A, Santos E, Jones J, Takeshita L, Ortega-Rivera ND, et al. Allele frequency net database (AFND) 2020 update: gold-standard data classification, open access genotype data and new query tools. Nucleic Acids Res. 2020;48:D783–d788.
Kersten B, Valkering M, Wouters R, van Amerongen R, Hanekamp D, Kwidama Z, et al. CD45RA, a specific marker for leukaemia stem cell sub-populations in acute myeloid leukaemia. Br J Haematol. 2016;173:219–35.
Hassan C, Kester MG, Oudgenoeg G, de Ru AH, Janssen GM, Drijfhout JW, et al. Accurate quantitation of MHC-bound peptides by application of isotopically labeled peptide MHC complexes. J Proteom. 2014;109:240–4.
Zeng AGX, Bansal S, Jin L, Mitchell A, Chen WC, Abbas HA, et al. A cellular hierarchy framework for understanding heterogeneity and predicting drug response in acute myeloid leukemia. Nat Med. 2022;28:1212–23.
Karakaslar EO, Severens JF, Sánchez-López E, van Veelen PA, Zlei M, van Dongen JJM, et al. A transcriptomic based deconvolution framework for assessing differentiation stages and drug responses of AML. NPJ Precis Oncol. 2024;8:105.
Sakurai M, Kunimoto H, Watanabe N, Fukuchi Y, Yuasa S, Yamazaki S, et al. Impaired hematopoietic differentiation of RUNX1-mutated induced pluripotent stem cells derived from FPD/AML patients. Leukemia. 2014;28:2344–54.
Schnittger S, Dicker F, Kern W, Wendland N, Sundermann J, Alpermann T, et al. RUNX1 mutations are frequent in de novo AML with noncomplex karyotype and confer an unfavorable prognosis. Blood. 2011;117:2348–57.
Costello R, Mallet F, Chambost H, Sainty D, Arnoulet C, Gastaut JA, et al. The immunophenotype of minimally differentiated acute myeloid leukemia (AML-M0): reduced immunogenicity and high frequency of CD34+/CD38- leukemic progenitors. Leukemia. 1999;13:1513–8.
Lapidot T, Sirard C, Vormoor J, Murdoch B, Hoang T, Caceres-Cortes J, et al. A cell initiating human acute myeloid leukaemia after transplantation into SCID mice. Nature. 1994;367:645–8.
Chopra M, Bohlander SK. The cell of origin and the leukemia stem cell in acute myeloid leukemia. Genes Chromosomes Cancer. 2019;58:850–8.
Sachs K, Sarver AL, Noble-Orcutt KE, LaRue RS, Antony ML, Chang D, et al. Single-cell gene expression analyses reveal distinct self-renewing and proliferating subsets in the leukemia stem cell compartment in acute myeloid leukemia. Cancer Res. 2020;80:458–70.
Lee JH, Shklovskaya E, Lim SY, Carlino MS, Menzies AM, Stewart A, et al. Transcriptional downregulation of MHC class I and melanoma de- differentiation in resistance to PD-1 inhibition. Nat Commun. 2020;11:1897.
Nagarsheth NB, Norberg SM, Sinkoe AL, Adhikary S, Meyer TJ, Lack JB, et al. TCR-engineered T cells targeting E7 for patients with metastatic HPV-associated epithelial cancers. Nat Med. 2021;27:419–25.
Montesion M, Murugesan K, Jin DX, Sharaf R, Sanchez N, Guria A, et al. Somatic HLA class I loss is a widespread mechanism of immune evasion which refines the use of tumor mutational burden as a biomarker of checkpoint inhibitor response. Cancer Discov. 2021;11:282–92.
Toffalori C, Zito L, Gambacorta V, Riba M, Oliveira G, Bucci G, et al. Immune signature drives leukemia escape and relapse after hematopoietic cell transplantation. Nat Med. 2019;25:603–11.
Pant S, Wainberg ZA, Weekes CD, Furqan M, Kasi PM, Devoe CE, et al. Lymph-node-targeted, mKRAS-specific amphiphile vaccine in pancreatic and colorectal cancer: the phase 1 AMPLIFY-201 trial. Nat Med. 2024;30:531–42.
Martinov T, Greenberg PD. Targeting driver oncogenes and other public neoantigens using T cell receptor-based cellular therapy. Annu Rev Cancer Biol. 2023;7:331–51.


















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