REFERENCES
1. Sung H, Ferlay J, Siegel RL, et al. Global cancer statistics 2020: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin 2021;71:209-49.
2. Llovet JM, Kelley RK, Villanueva A, et al. Hepatocellular carcinoma. Nat Rev Dis Primers 2021;7:6.
3. Kudo M, Finn RS, Qin S, et al. Lenvatinib versus sorafenib in first-line treatment of patients with unresectable hepatocellular carcinoma: a randomised phase 3 non-inferiority trial. Lancet 2018;391:1163-73.
4. Greten TF, Abou-Alfa GK, Cheng AL, et al. Society for immunotherapy of cancer (SITC) clinical practice guideline on immunotherapy for the treatment of hepatocellular carcinoma. J Immunother Cancer 2021;9:e002794.
5. Gallage S, García-Beccaria M, Szydlowska M, et al. The therapeutic landscape of hepatocellular carcinoma. Med 2021;2:505-52.
6. Lee MS, Ryoo BY, Hsu CH, et al. GO30140 investigators. Atezolizumab with or without bevacizumab in unresectable hepatocellular carcinoma (GO30140): an open-label, multicentre, phase 1b study. Lancet Oncol 2020;21:808-20.
7. Finn RS, Ryoo BY, Merle P, et al. KEYNOTE-240 investigators. Pembrolizumab As second-line therapy in patients with advanced hepatocellular carcinoma in KEYNOTE-240: a randomized, double-blind, phase III trial. J Clin Oncol 2020;38:193-202.
8. Finn RS, Qin S, Ikeda M, et al. IMbrave150 Investigators. Atezolizumab plus bevacizumab in unresectable hepatocellular carcinoma. N Engl J Med 2020;382:1894-905.
10. Kim YK. RNA therapy: rich history, various applications and unlimited future prospects. Exp Mol Med 2022;54:455-65.
12. Crooke ST, Witztum JL, Bennett CF, Baker BF. RNA-targeted therapeutics. Cell Metab 2018;27:714-39.
13. Wilson RC, Doudna JA. Molecular mechanisms of RNA interference. Annu Rev Biophys 2013;42:217-39.
14. Han X, Wang L, Li T, et al. Beyond blocking: engineering RNAi-mediated targeted immune checkpoint nanoblocker enables T-cell-independent cancer treatment. ACS Nano 2020;14:17524-34.
15. Zhang C, Zhao Y, Yang Y, et al. RNAi mediated silencing of Nanog expression suppresses the growth of human colorectal cancer stem cells. Biochem Biophys Res Commun 2021;534:254-60.
16. Pardi N, Hogan MJ, Porter FW, Weissman D. mRNA vaccines - a new era in vaccinology. Nat Rev Drug Discov 2018;17:261-79.
17. Rosenblum D, Gutkin A, Kedmi R, et al. CRISPR-Cas9 genome editing using targeted lipid nanoparticles for cancer therapy. Sci Adv 2020:6.
18. Adachi T, Nakamura Y. Aptamers: a review of their chemical properties and modifications for therapeutic application. Molecules 2019;24:4229.
19. Fire A, Xu S, Montgomery MK, Kostas SA, Driver SE, Mello CC. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 1998;391:806-11.
20. Martinez J, Patkaniowska A, Urlaub H, Lührmann R, Tuschl T. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell 2002;110:563-74.
21. Bernstein E, Caudy AA, Hammond SM, Hannon GJ. Role for a bidentate ribonuclease in the initiation step of RNA interference. Nature 2001;409:363-6.
22. Meister G, Tuschl T. Mechanisms of gene silencing by double-stranded RNA. Nature 2004;431:343-9.
23. Robb T, Reid G, Blenkiron C. Exploiting microRNAs as cancer therapeutics. Target Oncol 2017;12:163-78.
24. Roberts TC, Langer R, Wood MJA. Advances in oligonucleotide drug delivery. Nat Rev Drug Discov 2020;19:673-94.
25. Oura K, Morishita A, Masaki T. Molecular and functional roles of microRNAs in the progression of hepatocellular carcinoma-A review. Int J Mol Sci 2020;21:8362.
26. To KKW, Fong W, Tong CWS, Wu M, Yan W, Cho WCS. Advances in the discovery of microRNA-based anticancer therapeutics: latest tools and developments. Expert Opin Drug Discov 2020;15:63-83.
27. Barata P, Sood AK, Hong DS. RNA-targeted therapeutics in cancer clinical trials: Current status and future directions. Cancer Treat Rev 2016;50:35-47.
30. Mockey M, Gonçalves C, Dupuy FP, Lemoine FM, Pichon C, Midoux P. mRNA transfection of dendritic cells: synergistic effect of ARCA mRNA capping with poly(A) chains in cis and in trans for a high protein expression level. Biochem Biophys Res Commun 2006;340:1062-8.
31. Muttach F, Muthmann N, Rentmeister A. Synthetic mRNA capping. Beilstein J Org Chem 2017;13:2819-32.
32. Shanmugasundaram M, Senthilvelan A, Kore AR. Recent advances in modified cap analogs: synthesis, biochemical properties, and mRNA based vaccines. Chem Rec 2022;22:e202200005.
33. Orlandini von Niessen AG, Poleganov MA, Rechner C, et al. Improving mRNA-based therapeutic gene delivery by expression-augmenting 3' UTRs identified by cellular library screening. Mol Ther 2019;27:824-36.
34. Jia L, Mao Y, Ji Q, Dersh D, Yewdell JW, Qian SB. Decoding mRNA translatability and stability from the 5' UTR. Nat Struct Mol Biol 2020;27:814-21.
35. Alexaki A, Hettiarachchi GK, Athey JC, et al. Effects of codon optimization on coagulation factor IX translation and structure: Implications for protein and gene therapies. Sci Rep 2019;9:15449.
36. Karikó K, Weissman D. Naturally occurring nucleoside modifications suppress the immunostimulatory activity of RNA: implication for therapeutic RNA development. Curr Opin Drug Discov Devel 2007;10:523-32.
37. Holtkamp S, Kreiter S, Selmi A, et al. Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells. Blood 2006;108:4009-17.
38. Willis E, Pardi N, Parkhouse K, et al. Nucleoside-modified mRNA vaccination partially overcomes maternal antibody inhibition of de novo immune responses in mice. Sci Transl Med 2020:12.
39. Sharifnia Z, Bandehpour M, Kazemi B, Zarghami N. Design and development of modified mRNA encoding core antigen of hepatitis C virus: a possible application in vaccine production. Iran Biomed J 2019;23:57-67.
40. Karikó K, Muramatsu H, Welsh FA, et al. Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability. Mol Ther 2008;16:1833-40.
41. Oberli MA, Reichmuth AM, Dorkin JR, et al. Lipid nanoparticle assisted mRNA delivery for potent cancer immunotherapy. Nano Lett 2017;17:1326-35.
42. Chen J, Ye Z, Huang C, et al. Lipid nanoparticle-mediated lymph node-targeting delivery of mRNA cancer vaccine elicits robust CD8+ T cell response. Proc Natl Acad Sci U S A 2022;119:e2207841119.
43. Kreiter S, Vormehr M, van de Roemer N, et al. Mutant MHC class II epitopes drive therapeutic immune responses to cancer. Nature 2015;520:692-6.
44. Kranz LM, Diken M, Haas H, et al. Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy. Nature 2016;534:396-401.
45. Linares-Fernández S, Lacroix C, Exposito JY, Verrier B. Tailoring mRNA vaccine to balance innate/adaptive immune response. Trends Mol Med 2020;26:311-23.
46. Esprit A, de Mey W, Bahadur Shahi R, Thielemans K, Franceschini L, Breckpot K. Neo-antigen mRNA vaccines. Vaccines 2020;8:776.
47. Peng M, Mo Y, Wang Y, et al. Neoantigen vaccine: an emerging tumor immunotherapy. Mol Cancer 2019;18:128.
48. Keskin DB, Anandappa AJ, Sun J, et al. Neoantigen vaccine generates intratumoral T cell responses in phase Ib glioblastoma trial. Nature 2019;565:234-9.
49. Lang F, Schrörs B, Löwer M, Türeci Ö, Sahin U. Identification of neoantigens for individualized therapeutic cancer vaccines. Nat Rev Drug Discov 2022;21:261-82.
50. Liu JQ, Zhang C, Zhang X, et al. Intratumoral delivery of IL-12 and IL-27 mRNA using lipid nanoparticles for cancer immunotherapy. J Control Release 2022;345:306-13.
51. Tan AT, Meng F, Jin J, et al. Immunological alterations after immunotherapy with short lived HBV-TCR T cells associates with long-term treatment response in HBV-HCC. Hepatol Commun 2022;6:841-54.
52. Rajan T, Gugliandolo A, Bramanti P, Mazzon E. In vitro-transcribed mRNA chimeric antigen receptor T cell (IVT mRNA CAR T) therapy in hematologic and solid tumor management: a preclinical update. Int J Mol Sci 2020;21:6514.
54. Wu H, Lima WF, Zhang H, Fan A, Sun H, Crooke ST. Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs. J Biol Chem 2004;279:17181-9.
55. Liang XH, Sun H, Nichols JG, Crooke ST. RNase H1-dependent antisense oligonucleotides are robustly active in directing rna cleavage in both the cytoplasm and the nucleus. Mol Ther 2017;25:2075-92.
56. Ruhanen H, Ushakov K, Yasukawa T. Involvement of DNA ligase III and ribonuclease H1 in mitochondrial DNA replication in cultured human cells. Biochim Biophys Acta 2011;1813:2000-7.
57. Lima WF, Murray HM, Damle SS, et al. Viable RNaseH1 knockout mice show RNaseH1 is essential for R loop processing, mitochondrial and liver function. Nucleic Acids Res 2016;44:5299-312.
59. Cerritelli SM, Crouch RJ. RNases H: multiple roles in maintaining genome integrity. DNA Repair 2019;84:102742.
60. Lai F, Damle SS, Ling KK, Rigo F. Directed RNase H cleavage of nascent transcripts causes transcription termination. Mol Cell 2020;77:1032-1043.e4.
61. Khan P, Siddiqui JA, Lakshmanan I, et al. RNA-based therapies: a cog in the wheel of lung cancer defense. Mol Cancer 2021;20:54.
62. Crooke ST, Baker BF, Crooke RM, Liang XH. Antisense technology: an overview and prospectus. Nat Rev Drug Discov 2021;20:427-53.
63. Ramasamy T, Ruttala HB, Munusamy S, Chakraborty N, Kim JO. Nano drug delivery systems for antisense oligonucleotides (ASO) therapeutics. J Control Release 2022;352:861-78.
64. Desterro J, Bak-Gordon P, Carmo-Fonseca M. Targeting mRNA processing as an anticancer strategy. Nat Rev Drug Discov 2020;19:112-29.
65. Li D, Mastaglia FL, Fletcher S, Wilton SD. Precision medicine through antisense oligonucleotide-mediated exon skipping. trends Pharmacol Sci 2018;39:982-94.
68. Chen X, Mangala LS, Rodriguez-Aguayo C, Kong X, Lopez-Berestein G, Sood AK. RNA interference-based therapy and its delivery systems. Cancer Metastasis Rev 2018;37:107-24.
69. Meng C, Chen Z, Li G, Welte T, Shen H. Nanoplatforms for mRNA therapeutics. Adv Ther 2021;4:2000099.
70. Byun MJ, Lim J, Kim SN, et al. Advances in nanoparticles for effective delivery of RNA therapeutics. Biochip J 2022;16:128-45.
71. Li Y, Fang H, Zhang T, et al. Lipid-mRNA nanoparticles landscape for cancer therapy. Front Bioeng Biotechnol 2022;10:1053197.
72. Witzigmann D, Kulkarni JA, Leung J, Chen S, Cullis PR, van der Meel R. Lipid nanoparticle technology for therapeutic gene regulation in the liver. Adv Drug Deliv Rev 2020;159:344-63.
73. Hou X, Zaks T, Langer R, Dong Y. Lipid nanoparticles for mRNA delivery. Nat Rev Mater 2021;6:1078-94.
74. Gilleron J, Querbes W, Zeigerer A, et al. Image-based analysis of lipid nanoparticle-mediated siRNA delivery, intracellular trafficking and endosomal escape. Nat Biotechnol 2013;31:638-46.
75. Sato Y, Kinami Y, Hashiba K, Harashima H. Different kinetics for the hepatic uptake of lipid nanoparticles between the apolipoprotein E/low density lipoprotein receptor and the N-acetyl-d-galactosamine/asialoglycoprotein receptor pathway. J Control Release 2020;322:217-26.
76. Kamaly N, Xiao Z, Valencia PM, Radovic-Moreno AF, Farokhzad OC. Targeted polymeric therapeutic nanoparticles: design, development and clinical translation. Chem Soc Rev 2012;41:2971-3010.
77. Rai R, Alwani S, Badea I. Polymeric nanoparticles in gene therapy: new avenues of design and optimization for delivery applications. Polymers 2019;11:745.
79. Ke L, Cai P, Wu Y, Chen X. Polymeric nonviral gene delivery systems for cancer immunotherapy. Adv Ther 2020;3:1900213.
80. Aqil F, Munagala R, Jeyabalan J, et al. Milk exosomes - natural nanoparticles for siRNA delivery. Cancer Lett 2019;449:186-95.
81. Liang Y, Wang Y, Wang L, et al. Self-crosslinkable chitosan-hyaluronic acid dialdehyde nanoparticles for CD44-targeted siRNA delivery to treat bladder cancer. Bioact Mater 2021;6:433-46.
82. Höbel S, Appeldoorn CC, Gaillard PJ, Aigner A. Targeted CRM197-PEG-PEI/siRNA complexes for therapeutic RNAi in glioblastoma. Pharmaceuticals 2011;4:1591-606.
83. Pandey AP, Sawant KK. Polyethylenimine: a versatile, multifunctional non-viral vector for nucleic acid delivery. Mater Sci Eng C Mater Biol Appl 2016;68:904-18.
84. Shi YC, Zhao H, Yin C, et al. C/EBPα inhibits hepatocellular carcinoma by reducing Notch3/Hes1/p27 cascades. Dig Liver Dis 2013;45:844-51.
85. Voutila J, Reebye V, Roberts TC, et al. Development and mechanism of small activating RNA targeting CEBPA, a novel therapeutic in clinical trials for liver cancer. Mol Ther 2017;25:2705-14.
86. Sarker D, Plummer R, Meyer T, et al. MTL-CEBPA, a small activating RNA therapeutic upregulating C/EBP-α, in patients with advanced liver cancer: a first-in-human, multicenter, open-label, phase I trial. Clin Cancer Res 2020;26:3936-46.
87. Schaub FX, Dhankani V, Berger AC, et al. Cancer Genome Atlas Network. Pan-cancer alterations of the MYC oncogene and its proximal network across the cancer genome atlas. Cell Syst 2018;6:282-300.e2.
88. Liu F, Liao Z, Zhang Z. MYC in liver cancer: mechanisms and targeted therapy opportunities. Oncogene 2023;42:3303-18.
89. Liu X. Targeting polo-like kinases: a promising therapeutic approach for cancer treatment. Transl Oncol 2015;8:185-95.
90. Semple SC, Judge AD, Robbins M, et al. Abstract 2829: preclinical characterization of TKM-080301, a lipid nanoparticle formulation of a small interfering RNA directed against polo-like kinase 1. Cancer Res 2011;71:2829-2829.
91. El Dika I, Lim HY, Yong WP, et al. An open-label, multicenter, Phase I, dose escalation study with phase ii expansion cohort to determine the safety, pharmacokinetics, and preliminary antitumor activity of intravenous TKM-080301 in subjects with advanced hepatocellular carcinoma. Oncologist 2019;24:747-e218.
92. Cervantes A, Alsina M, Tabernero J, et al. Phase I dose-escalation study of ALN-VSP02, a novel RNAi therapeutic for solid tumors with liver involvement. JCO 2011;29:3025.
93. Zhang L, Liao Y, Tang L. MicroRNA-34 family: a potential tumor suppressor and therapeutic candidate in cancer. J Exp Clin Cancer Res 2019;38:53.
94. Hong DS, Kang YK, Borad M, et al. Phase 1 study of MRX34, a liposomal miR-34a mimic, in patients with advanced solid tumours. Br J Cancer 2020;122:1630-7.
95. Hu Z, Leet DE, Allesøe RL, et al. Personal neoantigen vaccines induce persistent memory T cell responses and epitope spreading in patients with melanoma. Nat Med 2021;27:515-25.
96. Weber JS, Carlino MS, Khattak A, et al. Individualised neoantigen therapy mRNA-4157 (V940) plus pembrolizumab versus pembrolizumab monotherapy in resected melanoma (KEYNOTE-942): a randomised, phase 2b study. Lancet 2024;403:632-44.
97. Rodriguez-rivera II, Wu T, Ciotti R, et al. A phase 1/2 open-label study to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of OTX-2002 as a single agent and in combination with standard of care in patients with hepatocellular carcinoma and other solid tumor types known for association with the MYC oncogene (MYCHELANGELO I). JCO 2023;41:TPS627-TPS627.
98. Oliver SE, Gargano JW, Marin M, et al. The advisory committee on immunization practices' interim recommendation for use of Pfizer-BioNTech COVID-19 vaccine - United States, December 2020. MMWR Morb Mortal Wkly Rep 2020;69:1922-4.
99. Ledford H. Moderna COVID vaccine becomes second to get US authorization. Nature 2020:Online ahead of print.
100. Childs-Disney JL, Yang X, Gibaut QMR, Tong Y, Batey RT, Disney MD. Targeting RNA structures with small molecules. Nat Rev Drug Discov 2022;21:736-62.
101. Falese JP, Donlic A, Hargrove AE. Targeting RNA with small molecules: from fundamental principles towards the clinic. Chem Soc Rev 2021;50:2224-43.
102. Winkle M, El-Daly SM, Fabbri M, Calin GA. Noncoding RNA therapeutics - challenges and potential solutions. Nat Rev Drug Discov 2021;20:629-51.
