Tomita A, Kiyoi H, Naoe T. Mechanisms of motion and resistance to all-trans retinoic acid (ATRA) and arsenic trioxide (As2O3) in acute promyelocytic leukemia. Int J Hematol. 2013;97:717–25.
Stubbins RJ, Karsan A. Differentiation remedy for myeloid malignancies: past cytotoxicity. Blood Most cancers J. 2021;11:193.
Stelmach P, Trumpp A. Leukemic stem cells and remedy resistance in acute myeloid leukemia. Haematologica. 2023;108:353–66.
de Thé H. Differentiation remedy revisited. Nat Rev Most cancers. 2018;18:117–27.
Rosenbauer F, Tenen DG. Transcription components in myeloid growth: balancing differentiation with transformation. Nat Rev Immunol. 2007;7:105–17.
Lu H, Zhou Q, He J, Jiang Z, Peng C, Tong R, et al. Current advances within the growth of protein–protein interactions modulators: mechanisms and scientific trials. Sign Transduct Goal Ther. 2020;5:213.
Dang CV, Reddy EP, Shokat KM, Soucek L. Drugging the ‘undruggable’ most cancers targets. Nat Rev Most cancers. 2017;17:502–8.
Kulkarni JA, Witzigmann D, Thomson SB, Chen S, Leavitt BR, Cullis PR, et al. The present panorama of nucleic acid therapeutics. Nat Nanotechnol. 2021;16:630–43.
Hearth A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and particular genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391:806–11.
Hu B, Zhong L, Weng Y, Peng L, Huang Y, Zhao Y, et al. Therapeutic siRNA: state-of-the-art. Sig Transduct Goal Ther. 2020;5:101.
Adams D, Gonzalez-Duarte A, O’Riordan WD, Yang C-C, Ueda M, Kristen AV, et al. Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis. N. Engl J Med. 2018;379:11–21.
Roberts TC, Langer R, Wooden MJA. Advances in oligonucleotide drug supply. Nat Rev Drug Discov. 2020;19:673–94.
Crooke ST, Liang X-H, Baker BF, Crooke RM Antisense know-how: a evaluation. J Biol Chem. 2021;296.
Khorkova O, Stahl J, Joji A, Volmar C-H, Wahlestedt C. Amplifying gene expression with RNA-targeted therapeutics. Nat Rev Drug Discov. 2023;22:539–61.
Ni S, Zhuo Z, Pan Y, Yu Y, Li F, Liu J, et al. Current progress in aptamer discoveries and modifications for therapeutic functions. ACS Appl Mater Interfaces. 2021;13:9500–19.
Yang S, Li H, Xu L, Deng Z, Han W, Liu Y, et al. Oligonucleotide aptamer-mediated precision remedy of hematological malignancies. Mol Ther Nucleic Acids. 2018;13:164–75.
Stuart RK, Stockerl-Goldstein Okay, Cooper M, Devetten M, Herzig R, Medeiros B, et al. Randomized part II trial of the nucleolin focusing on aptamer AS1411 mixed with high-dose cytarabine in relapsed/refractory acute myeloid leukemia (AML). J Clin Oncol. 2009;27:7019.
Tan CP, Sinigaglia L, Gomez V, Nicholls J, Habib NA. RNA activation—a novel method to therapeutically upregulate gene transcription. Molecules. 2021;26:6530.
Sarker D, Plummer R, Meyer T, Sodergren MH, Basu B, Chee CE, et al. MTL-CEBPA, a small activating RNA therapeutic upregulating C/EBP-α, in sufferers with superior liver most cancers: a first-in-human, multicenter, open-label, part I trial. Clin Most cancers Res. 2020;26:3936–46.
Hofman CR, Corey DR. Focusing on RNA with artificial oligonucleotides: scientific success invitations new challenges. Cell Chem Biol. 2024;31:125–38.
Egli M, Manoharan M. Chemistry, construction and performance of authorized oligonucleotide therapeutics. Nucleic Acids Res. 2023;51:2529–73.
Gait MJ, Agrawal S. Introduction and Historical past of the Chemistry of Nucleic Acids Therapeutics. Strategies Mol Biol. 2022;2434:3–31.
Goemans N, Mercuri E, Belousova E, Komaki H, Dubrovsky A, McDonald CM, et al. A randomized placebo-controlled part 3 trial of an antisense oligonucleotide, drisapersen, in Duchenne muscular dystrophy. Neuromuscul Disord. 2018;28:4–15.
Imbimbo BP, Triaca V, Imbimbo C, Nisticò R. Investigational remedies for neurodegenerative illnesses brought on by inheritance of gene mutations: classes from latest scientific trials. Neural Regen Res. 2023;18:1679–83.
Reed JC, Stein C, Subasinghe C, Haldar S, Croce CM, Yum S, et al. Antisense-mediated inhibition of BCL2 protooncogene expression and leukemic cell development and survival: comparisons of phosphodiester and phosphorothioate oligodeoxynucleotides. Most cancers Res. 1990;50:6565–70.
Walker AR, Marcucci G, Yin J, Blum W, Inventory W, Kohlschmidt J, et al. Section 3 randomized trial of chemotherapy with or with out oblimersen in older AML sufferers: CALGB 10201 (Alliance). Blood Adv. 2021;5:2775–87.
Winkler J, Stessl M, Amartey J, Noe CR. Off-target results associated to the phosphorothioate modification of nucleic acids. ChemMedChem 2010;5:1344–52.
Tenchov R, Chook R, Curtze AE, Zhou Q. Lipid nanoparticles─from liposomes to mRNA vaccine supply, a panorama of analysis variety and development. ACS Nano. 2021;15:16982–7015.
Chander N, Basha G, Yan Cheng MH, Witzigmann D, Cullis PR. Lipid nanoparticle mRNA methods containing excessive ranges of sphingomyelin engender larger protein expression in hepatic and extra-hepatic tissues. Mol Ther Methodology Clin Develop. 2023;30:235–45.
Jung HN, Lee SY, Lee S, Youn H, Im HJ. Lipid nanoparticles for supply of RNA therapeutics: present standing and the function of in vivo imaging. Theranostics 2022;12:7509–31.
Paunovska Okay, Loughrey D, Dahlman JE. Drug supply methods for RNA therapeutics. Nat Rev Genet. 2022;23:265–80.
Dowdy SF, Setten RL, Cui XS, Jadhav SG. Supply of RNA therapeutics: the nice endosomal escape! Nucleic Acid Ther. 2022;32:361–8.
Vago L, Gojo I. Immune escape and immunotherapy of acute myeloid leukemia. J Clin Investig. 2020;130:1552–64.
Shao R, Li Z, Xin H, Jiang S, Zhu Y, Liu J, et al. Biomarkers as targets for CAR-T/NK cell remedy in AML. Biomarker Res. 2023;11:65.
Mercier FE, Ragu C, Scadden DT. The bone marrow on the crossroads of blood and immunity. Nat Rev Immunol. 2012;12:49–60.
Estey E, Levine RL, Löwenberg B. Present challenges in scientific growth of “focused therapies”: the case of acute myeloid leukemia. Blood. 2015;125:2461–6.
Alagpulinsa DA, Toribio MP, Alhallak I, Shmookler Reis RJ. Advances in understanding the molecular foundation of clonal hematopoiesis. Tendencies Mol Med. 2022;28:360–77.
Docking TR, Parker JDK, Jädersten M, Duns G, Chang L, Jiang J, et al. A scientific transcriptome method to affected person stratification and remedy choice in acute myeloid leukemia. Nat Commun. 2021;12:2474.
Reikvam H, Hatfield KJ, Kittang AO, Hovland R, Bruserud Ø. Acute myeloid leukemia with the t(8;21) translocation: scientific penalties and organic implications. J Biomed Biotechnol. 2011;2011:104631.
Pabst T, Mueller BU, Harakawa N, Schoch C, Haferlach T, Behre G, et al. AML1-ETO downregulates the granulocytic differentiation issue C/EBPalpha in t(8;21) myeloid leukemia. Nat Med. 2001;7:444–51.
Issa H, Swart LE, Rasouli M, Ashtiani M, Nakjang S, Jyotsana N, et al. Nanoparticle-mediated focusing on of the fusion gene RUNX1/ETO in t(8;21)-positive acute myeloid leukaemia. Leukemia 2023;37:820–34.
Ptasinska A, Assi SA, Mannari D, James SR, Williamson D, Dunne J, et al. Depletion of RUNX1/ETO in t(8;21) AML cells results in genome-wide adjustments in chromatin construction and transcription issue binding. Leukemia. 2012;26:1829–41.
Heidenreich O, Krauter J, Riehle H, Hadwiger P, John M, Heil G, et al. AML1/MTG8 oncogene suppression by small interfering RNAs helps myeloid differentiation of t(8;21)-positive leukemic cells. Blood. 2003;101:3157–63.
Cilloni D, Saglio G. Molecular pathways: BCR-ABL. Clin Most cancers Res. 2012;18:930–7.
Wilda M, Fuchs U, Wössmann W, Borkhardt A. Killing of leukemic cells with a BCR/ABL fusion gene by RNA interference (RNAi). Oncogene. 2002;21:5716–24.
Remant KC, Thapa B, Valencia-Serna J, Domun SS, Dimitroff C, Jiang X, et al. Ldl cholesterol grafted cationic lipopolymers: Potential siRNA carriers for selective power myeloid leukemia remedy. J Biomed Mater Res A. 2020;108:565–80.
Yang C, Panwar N, Wang Y, Zhang B, Liu M, Toh H, et al. Biodegradable charged polyester-based vectors (BCPVs) as an environment friendly non-viral transfection nanoagent for gene knockdown of the BCR–ABL hybrid oncogene in a human power myeloid leukemia cell line. Nanoscale. 2016;8:9405–16.
Zhang X, Koh CG, Yu B, Liu S, Piao L, Marcucci G, et al. Transferrin receptor focused lipopolyplexes for supply of antisense oligonucleotide g3139 in a murine k562 xenograft mannequin. Pharm Res. 2009;26:1516–24.
Valencia-Serna J, Aliabadi HM, Manfrin A, Mohseni M, Jiang X, Uludag H. siRNA/lipopolymer nanoparticles to arrest development of power myeloid leukemia cells in vitro and in vivo. Eur J Pharm Biopharm. 2018;130:66–70.
Letai A, Sorcinelli MD, Beard C, Korsmeyer SJ. Antiapoptotic BCL-2 is required for upkeep of a mannequin leukemia. Most cancers Cell. 2004;6:241–9.
Ong F, Kim Okay, Konopleva MY. Venetoclax resistance: mechanistic insights and future methods. Most cancers Drug Resist. 2022;5:380–400.
Gutiérrez-Puente Y, Tari AM, Stephens C, Rosenblum M, Guerra RT, Lopez-Berestein G. Security, pharmacokinetics, and tissue distribution of liposomal p-ethoxy antisense oligonucleotides focused to Bcl-2. J Pharmacol Exp Ther. 1999;291:865–9.
Summary from Proceedings of the American Affiliation for Most cancers Analysis Annual Assembly 2021 Gagliardi M, Ashizawa AT The mix of liposomal Bcl-2 antisense oligonucleotide (BP1002) with decitabine is efficacious in venetoclax-resistant cells. Most cancers Analysis. 2021;81:Summary nr 939.
Maiti A, Carter BZ, Andreeff M, Konopleva MY. SOHO state-of-the-art updates and subsequent questions | Past BCL-2 inhibition in acute myeloid leukemia: different approaches to leverage the apoptotic pathway. Clin Lymphoma Myeloma Leuk. 2022;22:652–8.
Widden H, Placzek WJ. The a number of mechanisms of MCL1 within the regulation of cell destiny. Commun Biol. 2021;4:1029.
Steimer DA, Boyd Okay, Takeuchi O, Fisher JK, Zambetti GP, Opferman JT. Selective roles for antiapoptotic MCL-1 throughout granulocyte growth and macrophage effector operate. Blood. 2009;113:2805–15.
Wan Y, Dai N, Tang Z, Fang H. Small-molecule Mcl-1 inhibitors: rising anti-tumor brokers. Eur J Medicinal Chem. 2018;146:471–82.
Yang J, Ikezoe T, Nishioka C, Yokoyama A. Over-expression of Mcl-1 impairs the flexibility of ATRA to induce development arrest and differentiation in acute promyelocytic leukemia cells. Apoptosis. 2013;18:1403–15.
Karami H, Baradaran B, Esfahani A, Sakhinia M, Sakhinia E. Therapeutic results of myeloid cell leukemia-1 siRNA on human acute myeloid leukemia cells. Adv Pharm Bull. 2014;4:243–8.
Yoshimoto G, Miyamoto T, Jabbarzadeh-Tabrizi S, Iino T, Rocnik JL, Kikushige Y, et al. FLT3-ITD up-regulates MCL-1 to advertise survival of stem cells in acute myeloid leukemia by way of FLT3-ITD-specific STAT5 activation. Blood. 2009;114:5034–43.
Fernandez S, Desplat V, Villacreces A, Guitart AV, Milpied N, Pigneux A, et al. Focusing on tyrosine kinases in acute myeloid leukemia: why, who and the way? Int J Mol Sci. 2019;20:3429.
Tari AM, Hung M-C, Li Okay, Lopez-Berestein G. Progress inhibition of breast most cancers cells by Grb2 downregulation is correlated with inactivation of mitogen-activated protein kinase in EGFR, however not in ErbB2, cells. Oncogene. 1999;18:1325–32.
Tari AM, Gutiérrez-Puente Y, Monaco G, Stephens C, Solar T, Rosenblum M, et al. Liposome-incorporated Grb2 antisense oligodeoxynucleotide will increase the survival of mice bearing bcr-abl-positive leukemia xenografts. Int J Oncol. 2007;31:1243–50.
Ohanian M, Tari Ashizawa A, Garcia-Manero G, Pemmaraju N, Kadia T, Jabbour E, et al. Liposomal Grb2 antisense oligodeoxynucleotide (BP1001) in sufferers with refractory or relapsed haematological malignancies: a single-centre, open-label, dose-escalation, part 1/1b trial. Lancet Haematol. 2018;5:e136–e146.
Velu CS, Chaubey A, Phelan JD, Horman SR, Wunderlich M, Guzman ML, et al. Therapeutic antagonists of microRNAs deplete leukemia-initiating cell exercise. J Clin Make investments. 2014;124:222–36.
Fei J, Li Y, Zhu X, Luo X. miR-181a post-transcriptionally downregulates oncogenic RalA and contributes to development inhibition and apoptosis in power myelogenous leukemia (CML). PLoS One. 2012;7:e32834.
Su R, Li C, Wang X, Li Z, Wen Z, Yin Z, et al. PPFIA1-targeting miR-181a mimic and saRNA overcome imatinib resistance in BCR-ABL1-independent power myeloid leukemia by suppressing leukemia stem cell regeneration. Mol Ther Nucleic Acids. 2023;32:729–42.
Shastri A, Choudhary G, Teixeira M, Gordon-Mitchell S, Ramachandra N, Bernard L, et al. Antisense STAT3 inhibitor decreases viability of myelodysplastic and leukemic stem cells. J Clin Make investments. 2018;128:5479–88.
Hong D, Kurzrock R, Kim Y, Woessner R, Younes A, Nemunaitis J, et al. AZD9150, a next-generation antisense oligonucleotide inhibitor of STAT3 with early proof of scientific exercise in lymphoma and lung most cancers. Sci Transl Med. 2015;7:314ra185.
Hossain DM, Dos Santos C, Zhang Q, Kozlowska A, Liu H, Gao C, et al. Leukemia cell-targeted STAT3 silencing and TLR9 triggering generate systemic antitumor immunity. Blood. 2014;123:15–25.
Kurtz KJ, Conneely SE, O’Keefe M, Wohlan Okay, Rau RE. Murine fashions of acute myeloid leukemia. Entrance Oncol. 2022;12:854973.
Mazurek A, Park Y, Miething C, Wilkinson JE, Gillis J, Lowe SW, et al. Acquired dependence of acute myeloid leukemia on the DEAD-box RNA helicase DDX5. Cell Rep. 2014;7:1887–99.
Wu J, You YQ, Ma YX, Kang YH, Wu T, Wu XJ, et al. DDX5-targeting absolutely human monoclonal autoantibody inhibits proliferation and promotes differentiation of acute promyelocytic leukemia cells by rising ROS manufacturing. Cell Demise Dis. 2020;11:552.
Le TK, Cherif C, Omabe Okay, Paris C, Lannes F, Audebert S, et al. DDX5 mRNA-targeting antisense oligonucleotide as a brand new promising therapeutic in combating castration-resistant prostate most cancers. Mol Ther. 2023;31:471–86.
Liu J, Tong J, Yang H. Focusing on CD33 for acute myeloid leukemia remedy. BMC Most cancers. 2022;22:24.
Hansel TT, Kropshofer H, Singer T, Mitchell JA, George AJT. The security and uncomfortable side effects of monoclonal antibodies. Nat Rev Drug Discov. 2010;9:325–38.
Yang C, Wang Y, Ge MH, Fu YJ, Hao R, Islam Okay, et al. Fast identification of particular DNA aptamers exactly focusing on CD33 constructive leukemia cells by way of a paired cell-based method. Biomater Sci. 2019;7:938–50.
Soundararajan S, Wang L, Sridharan V, Chen W, Courtenay-Luck N, Jones D, et al. Plasma membrane nucleolin is a receptor for the anticancer aptamer AS1411 in MV4-11 leukemia cells. Mol Pharm. 2009;76:984–91.
Wang M, Wu H, Duan M, Yang Y, Wang G, Che F, et al. SS30, a novel thioaptamer focusing on CD123, inhibits the expansion of acute myeloid leukemia cells. Life Sci. 2019;232:116663.
Zhao N, Pei S-N, Qi J, Zeng Z, Iyer SP, Lin P, et al. Oligonucleotide aptamer-drug conjugates for focused remedy of acute myeloid leukemia. Biomaterials. 2015;67:42–51.
Suzuki H, Forrest AR, van Nimwegen E, Daub CO, Balwierz PJ, Irvine KM, et al. The transcriptional community that controls development arrest and differentiation in a human myeloid leukemia cell line. Nat Genet. 2009;41:553–62.
Wang E, Zhou H, Nadorp B, Cayanan G, Chen X, Yeaton AH, et al. Floor antigen-guided CRISPR screens establish regulators of myeloid leukemia differentiation. Cell Stem Cell. 2021;28:718–.e16.
Tzelepis Okay, Koike-Yusa H, De Braekeleer E, Li Y, Metzakopian E, Dovey OM, et al. A CRISPR dropout display screen identifies genetic vulnerabilities and therapeutic targets in acute myeloid leukemia. Cell Rep. 2016;17:1193–205.
Gourvest M, Brousset P, Bousquet M. Lengthy noncoding RNAs in acute myeloid leukemia: useful characterization and scientific relevance. Cancers (Basel). 2019;11:1638.
Bi L, Solar L, Jin Z, Zhang S, Shen Z. MicroRNA-10a/b are regulators of myeloid differentiation and acute myeloid leukemia. Oncol Lett. 2018;15:5611–9.
Wallace JA, O’Connell RM. MicroRNAs and acute myeloid leukemia: therapeutic implications and rising ideas. Blood. 2017;130:1290–301.
Gong JN, Yu J, Lin HS, Zhang XH, Yin XL, Xiao Z, et al. The function, mechanism and probably therapeutic software of microRNA-29 household in acute myeloid leukemia. Cell Demise Differ. 2014;21:100–12.
Singh V, Uddin MH, Zonder JA, Azmi AS, Balasubramanian SK. Round RNAs in acute myeloid leukemia. Mol Most cancers. 2021;20:149.
Usman WM, Pham TC, Kwok YY, Vu LT, Ma V, Peng B, et al. Environment friendly RNA drug supply utilizing purple blood cell extracellular vesicles. Nat Commun. 2018;9:2359.
Guimarães PPG, Figueroa-Espada CG, Riley RS, Gong N, Xue L, Sewastianik T, et al. In vivo bone marrow microenvironment siRNA supply utilizing lipid–polymer nanoparticles for a number of myeloma remedy. Proc Natl Acad Sci. 2023;120:e2215711120.
Halley PD, Lucas CR, McWilliams EM, Webber MJ, Patton RA, Kural C, et al. Daunorubicin-loaded DNA origami nanostructures circumvent drug-resistance mechanisms in a leukemia mannequin. Small. 2016;12:308–20.
Sefah Okay, Tang ZW, Shangguan DH, Chen H, Lopez-Colon D, Li Y, et al. Molecular recognition of acute myeloid leukemia utilizing aptamers. Leukemia. 2009;23:235–44.
Chung SS, Eng WS, Hu W, Khalaj M, Garrett-Bakelman FE, Tavakkoli M, et al. CD99 is a therapeutic goal on illness stem cells in myeloid malignancies. Sci Transl Med. 2017;9:eaaj2025.
Krupka C, Lichtenegger FS, Köhnke T, Bögeholz J, Bücklein V, Roiss M, et al. Focusing on CD157 in AML utilizing a novel, Fc-engineered antibody assemble. Oncotarget.2017;8:35707–17.
Haubner S, Perna F, Köhnke T, Schmidt C, Berman S, Augsberger C, et al. Coexpression profile of leukemic stem cell markers for combinatorial focused remedy in AML. Leukemia. 2019;33:64–74.
Fakhr E, Zare F, Teimoori-Toolabi L. Exact and environment friendly siRNA design: a key level in competent gene silencing. Most cancers Gene Ther. 2016;23:73–82.
Partridge W, Xia S, Kwoh TJ, Bhanot S, Geary RS, Baker BF. Enhancements within the tolerability profile of two′-O-methoxyethyl chimeric antisense oligonucleotides in parallel with advances in design. Display screen Different Strategies Nucleic Acid Ther. 2021;31:417–26.
Wang J, Place RF, Portnoy V, Huang V, Kang MR, Kosaka M, et al. Inducing gene expression by focusing on promoter sequences utilizing small activating RNAs. J Biol Strategies. 2015;2:e14.
Sefah Okay, Shangguan D, Xiong X, O’Donoghue MB, Tan W. Improvement of DNA aptamers utilizing Cell-SELEX. Nat Protoc. 2010;5:1169–85.
Goyenvalle A, Jimenez-Mallebrera C, van Roon W, Stitching S, Krieg AM, Arechavala-Gomeza V, et al. Issues within the preclinical evaluation of the protection of antisense oligonucleotides. Nucleic Acid Ther. 2023;33:1–16.
