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| Advance of microRNA Therapeutics in Cancer and Other Diseases |
TANG De-ping1,XING Meng-jie1,2,SONG Wen-tao1,YAO Hui-hui1,MAO Ai-hong2,**( ) |
1 School of Biological & Pharmaceutical Engineering, Lanzhou Jiaotong University, Lanzhou 730070,China
2 Gansu Provincial Academic Institute for Medical Research, Lanzhou 730050,China |
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Abstract microRNAs (miRNAs) are a class of endogenous, small non-coding RNAs (about 22 nt) that have critical roles in gene expression. Functional studies have identified miRNAs dysregulation as a cause in many human diseases, including cancer, viral infection, and autoimmune disorders. Restoring or suppressing miRNAs function and activity has become an attractive therapeutic method for the management of cancer and other diseases. Several miRNA-based therapeutics have reached clinical development, including a mimic of the tumor suppressor miR-34 for treating cancer and anti-miRs targeted miR-122 for treating HCV infection. In this review, we summarize the recent advances in our knowledge of miRNA therapeutics in cancer and other diseases and discuss ongoing challenges to ensure the safety and efficacy of miRNA therapeutics in vivo.
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Received: 15 June 2021
Published: 01 December 2021
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Corresponding Authors:
Ai-hong MAO
E-mail: maoaih@aliyun.com
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| [1] |
Iorio M V, Croce C M. MicroRNA dysregulation in cancer: diagnostics, monitoring and therapeutics. A comprehensive review. EMBO Molecular Medicine, 2012, 4(3):143-159.
doi: 10.1002/emmm.v4.3
|
|
|
| [2] |
Baek J, Kang S, Min H. MicroRNA-targeting therapeutics for hepatitis C. Archives of Pharmacal Research, 2014, 37(3):299-305.
doi: 10.1007/s12272-013-0318-9
|
|
|
| [3] |
Gurha P. MicroRNAs in cardiovascular disease. Current Opinion in Cardiology, 2016, 31(3):249-254.
doi: 10.1097/HCO.0000000000000280
|
|
|
| [4] |
Rupaimoole R, Slack F J. MicroRNA therapeutics: towards a new era for the management of cancer and other diseases. Nature Reviews Drug Discovery, 2017, 16(3):203-222.
doi: 10.1038/nrd.2016.246
pmid: 28209991
|
|
|
| [5] |
Li Z H, Rana T M. Therapeutic targeting of microRNAs: current status and future challenges. Nature Reviews Drug Discovery, 2014, 13(8):622-638.
doi: 10.1038/nrd4359
|
|
|
| [6] |
To K K W, Fong W, Tong C W S, et al. Advances in the discovery of microRNA-based anticancer therapeutics: latest tools and developments. Expert Opinion on Drug Discovery, 2020, 15(1):63-83.
doi: 10.1080/17460441.2020.1690449
|
|
|
| [7] |
Okada N, Lin C P, Ribeiro M C, et al. A positive feedback between p53 and miR-34 miRNAs mediates tumor suppression. Genes & Development, 2014, 28(5):438-450.
doi: 10.1101/gad.233585.113
|
|
|
| [8] |
Wang X, Li J G, Dong K, et al. Tumor suppressor miR-34a targets PD-L1 and functions as a potential immunotherapeutic target in acute myeloid leukemia. Cellular Signalling, 2015, 27(3):443-452.
doi: 10.1016/j.cellsig.2014.12.003
pmid: 25499621
|
|
|
| [9] |
Cortez M A, Ivan C, Valdecanas D, et al. PDL1 Regulation by p53 via miR-34. JNCI: Journal of the National Cancer Institute, 2016, 108(1). DOI: 10.1093/jnci/djv303.
doi: 10.1093/jnci/djv303
|
|
|
| [10] |
Wiggins J F, Ruffino L, Kelnar K, et al. Development of a lung cancer therapeutic based on the tumor suppressor microRNA-34. Cancer Research, 2010, 70(14):5923-5930.
doi: 10.1158/0008-5472.CAN-10-0655
pmid: 20570894
|
|
|
| [11] |
Trang P, Wiggins J F, Daige C L, et al. Systemic delivery of tumor suppressor microRNA mimics using a neutral lipid emulsion inhibits lung tumors in mice. Molecular Therapy, 2011, 19(6):1116-1122.
doi: 10.1038/mt.2011.48
|
|
|
| [12] |
Kasinski A L, Slack F J. miRNA-34 prevents cancer initiation and progression in a therapeutically resistant K-ras and p53-induced mouse model of lung adenocarcinoma. Cancer Research, 2012, 72(21):5576-5587.
doi: 10.1158/0008-5472.CAN-12-2001
pmid: 22964582
|
|
|
| [13] |
Pramanik D, Campbell N R, Karikari C, et al. Restitution of tumor suppressor microRNAs using a systemic nanovector inhibits pancreatic cancer growth in mice. Molecular Cancer Therapeutics, 2011, 10(8):1470-1480.
doi: 10.1158/1535-7163.MCT-11-0152
|
|
|
| [14] |
Liu C, Kelnar K, Liu B G, et al. The microRNA miR-34a inhibits prostate cancer stem cells and metastasis by directly repressing CD44. Nature Medicine, 2011, 17(2):211-215.
doi: 10.1038/nm.2284
|
|
|
| [15] |
Gregory P A, Bert A G, Paterson E L, et al. The miR-200 family and miR-205 regulate epithelial to mesenchymal transition by targeting ZEB1 and SIP1. Nature Cell Biology, 2008, 10(5):593-601.
doi: 10.1038/ncb1722
pmid: 18376396
|
|
|
| [16] |
Korpal M, Lee E S, Hu G H, et al. The miR-200 family inhibits epithelial-mesenchymal transition and cancer cell migration by direct targeting of E-cadherin transcriptional repressors ZEB1 and ZEB2. Journal of Biological Chemistry, 2008, 283(22):14910-14914.
doi: 10.1074/jbc.C800074200
pmid: 18411277
|
|
|
| [17] |
Gregory P A, Bracken C P, Smith E, et al. An autocrine TGF-beta/ZEB/miR-200 signaling network regulates establishment and maintenance of epithelial-mesenchymal transition. Molecular Biology of the Cell, 2011, 22(10):1686-1698.
doi: 10.1091/mbc.E11-02-0103
pmid: 21411626
|
|
|
| [18] |
Pecot C V, Rupaimoole R, Yang D, et al. Tumour angiogenesis regulation by the miR-200 family. Nature Communications, 2013, 4:2427.
doi: 10.1038/ncomms3427
pmid: 24018975
|
|
|
| [19] |
Cortez M A, Valdecanas D, Zhang X C, et al. Therapeutic delivery of miR-200c enhances radiosensitivity in lung cancer. Molecular Therapy, 2014, 22(8):1494-1503.
doi: 10.1038/mt.2014.79
|
|
|
| [20] |
Bahreyni A, Rezaei M, Bahrami A, et al. Diagnostic, prognostic, and therapeutic potency of microRNA 21 in the pathogenesis of colon cancer, current status and prospective. Journal of Cellular Physiology, 2019, 234(6):8075-8081.
doi: 10.1002/jcp.v234.6
|
|
|
| [21] |
Wang Y, Zhou S Y, Fan K F, et al. MicroRNA-21 and its impact on signaling pathways in cervical cancer. Oncology Letters, 2019, 17(3):3066-3070.
|
|
|
| [22] |
Sekar D, Mani P, Biruntha M, et al. Dissecting the functional role of microRNA 21 in osteosarcoma. Cancer Gene Therapy, 2019, 26(7):179-182.
doi: 10.1038/s41417-019-0092-z
|
|
|
| [23] |
Medina P P, Nolde M, Slack F J. OncomiR addiction in an in vivo model of microRNA-21-induced pre-B-cell lymphoma. Nature, 2010, 467(7311):86-90.
doi: 10.1038/nature09284
|
|
|
| [24] |
Hatley M E, Patrick D M, Garcia M R, et al. Modulation of K-ras-dependent lung tumorigenesis by MicroRNA-21. Cancer Cell, 2010, 18(3):282-293.
doi: 10.1016/j.ccr.2010.08.013
pmid: 20832755
|
|
|
| [25] |
Krichevsky A M, Gabriely G. miR-21: a small multi-faceted RNA. Journal of Cellular and Molecular Medicine, 2008, 13(1):39-53.
doi: 10.1111/j.1582-4934.2008.00556.x
|
|
|
| [26] |
Campbell J D, Alexandrov A, Kim J, et al. Distinct patterns of somatic genome alterations in lung adenocarcinomas and squamous cell carcinomas. Nature Genetics, 2016, 48(6):607-616.
doi: 10.1038/ng.3564
pmid: 27158780
|
|
|
| [27] |
Saheb Sharif-Askari N, Saheb Sharif-Askari F, Guraya S Y, et al. Integrative systematic review meta-analysis and bioinformatics identifies MicroRNA-21 and its target genes as biomarkers for colorectal adenocarcinoma. International Journal of Surgery, 2020, 73:113-122.
doi: S1743-9191(19)30338-3
pmid: 31756546
|
|
|
| [28] |
Javanmardi S, Aghamaali M R, Abolmaali S S, et al. miR-21, an oncogenic target miRNA for cancer therapy: molecular mechanisms and recent advancements in chemo and radio-resistance. Current Gene Therapy, 2017, 16(6):375-389.
doi: 10.2174/1566523217666170102105119
pmid: 28042781
|
|
|
| [29] |
Jiang S, Zhang H W, Lu M H, et al. MicroRNA-155 functions as an OncomiR in breast cancer by targeting the suppressor of cytokine signaling 1 gene. Cancer Research, 2010, 70(8):3119-3127.
doi: 10.1158/0008-5472.CAN-09-4250
|
|
|
| [30] |
Chang S, Wang R H, Akagi K, et al. Tumor suppressor BRCA1 epigenetically controls oncogenic microRNA-155. Nature Medicine, 2011, 17(10):1275-1282.
doi: 10.1038/nm.2459
|
|
|
| [31] |
Due H, Schönherz A A, Ryø L, et al. MicroRNA-155 controls vincristine sensitivity and predicts superior clinical outcome in diffuse large B-cell lymphoma. Blood Advances, 2019, 3(7):1185-1196.
doi: 10.1182/bloodadvances.2018029660
|
|
|
| [32] |
Tili E, Michaille J J, Wernicke D, et al. Mutator activity induced by microRNA-155 (miR-155) links inflammation and cancer. PNAS, 2011, 108(12):4908-4913.
doi: 10.1073/pnas.1101795108
|
|
|
| [33] |
Kong W, He L, Richards E J, et al. Upregulation of miRNA-155 promotes tumour angiogenesis by targeting VHL and is associated with poor prognosis and triple-negative breast cancer. Oncogene, 2014, 33(6):679-689.
doi: 10.1038/onc.2012.636
pmid: 23353819
|
|
|
| [34] |
Babar I A, Cheng C J, Booth C J, et al. Nanoparticle-based therapy in an in vivo microRNA-155 (miR-155)-dependent mouse model of lymphoma. PNAS, 2012, 109(26):E1695-E1704. DOI: 10.1073/pnas.1201516109.
doi: 10.1073/pnas.1201516109
|
|
|
| [35] |
Cheng C J, Bahal R, Babar I A, et al. MicroRNA silencing for cancer therapy targeted to the tumour microenvironment. Nature, 2015, 518(7537):107-110.
doi: 10.1038/nature13905
|
|
|
| [36] |
Sarnow P, Sagan S M. Unraveling the mysterious interactions between hepatitis C virus RNA and liver-specific MicroRNA-122. Annual Review of Virology, 2016, 3(1):309-332.
pmid: 27578438
|
|
|
| [37] |
Luna J M, Scheel T K H, Danino T, et al. Hepatitis C virus RNA functionally sequesters miR-122. Cell, 2015, 160(6):1099-1110.
doi: 10.1016/j.cell.2015.02.025
|
|
|
| [38] |
Elmén J, Lindow M, Silahtaroglu A, et al. Antagonism of microRNA-122 in mice by systemically administered LNA-antimiR leads to up-regulation of a large set of predicted target mRNAs in the liver. Nucleic Acids Research, 2008, 36(4):1153-1162.
doi: 10.1093/nar/gkm1113
|
|
|
| [39] |
Tsai W C, Hsu S D, Hsu C S, et al. MicroRNA-122 plays a critical role in liver homeostasis and hepatocarcinogenesis. The Journal of Clinical Investigation, 2012, 122(8):2884-2897.
doi: 10.1172/JCI63455
|
|
|
| [40] |
Elmén J, Lindow M, Schütz S, et al. LNA-mediated microRNA silencing in non-human Primates. Nature, 2008, 452(7189):896-899.
doi: 10.1038/nature06783
|
|
|
| [41] |
Bejerano T, Etzion S, Elyagon S, et al. Nanoparticle delivery of miRNA-21 mimic to cardiac macrophages improves myocardial remodeling after myocardial infarction. Nano Letters, 2018, 18(9):5885-5891.
doi: 10.1021/acs.nanolett.8b02578
pmid: 30141949
|
|
|
| [42] |
Xin M, Small E M, Sutherland L B, et al. MicroRNAs miR-143 and miR-145 modulate cytoskeletal dynamics and responsiveness of smooth muscle cells to injury. Genes & Development, 2009, 23(18):2166-2178.
doi: 10.1101/gad.1842409
|
|
|
| [43] |
Ikeda S, He A B, Kong S W, et al. MicroRNA-1 negatively regulates expression of the hypertrophy-associated calmodulin and Mef2a genes. Molecular and Cellular Biology, 2009, 29(8):2193-2204.
doi: 10.1128/MCB.01222-08
|
|
|
| [44] |
Shan Z X, Lin Q X, Fu Y H, et al. Upregulated expression of miR-1/miR-206 in a rat model of myocardial infarction. Biochemical and Biophysical Research Communications, 2009, 381(4):597-601.
doi: 10.1016/j.bbrc.2009.02.097
|
|
|
| [45] |
Thanikachalam P V, Ramamurthy S, Wong Z W, et al. Current attempts to implement microRNA-based diagnostics and therapy in cardiovascular and metabolic disease: a promising future. Drug Discovery Today, 2018, 23(3):460-480.
doi: S1359-6446(17)30222-2
pmid: 29107764
|
|
|
| [46] |
Price N L, Miguel V, Ding W, et al. Genetic deficiency or pharmacological inhibition of miR-33 protects from kidney fibrosis. JCI Insight, 2019, 4(22). DOI: 10.1172/jci.insight.131102.
doi: 10.1172/jci.insight.131102
|
|
|
| [47] |
Rayner K J, Esau C C, Hussain F N, et al. Inhibition of miR-33a/b in non-human Primates raises plasma HDL and lowers VLDL triglycerides. Nature, 2011, 478(7369):404-407.
doi: 10.1038/nature10486
|
|
|
| [48] |
Goedeke L, Salerno A, Ramírez C M, et al. Long-term therapeutic silencing of miR-33 increases circulating triglyceride levels and hepatic lipid accumulation in mice. EMBO Molecular Medicine, 2014, 6(9):1133-1141.
doi: 10.15252/emmm.201404046
pmid: 25038053
|
|
|
| [49] |
Belgardt B F, Ahmed K, Spranger M, et al. The microRNA-200 family regulates pancreatic beta cell survival in type 2 diabetes. Nature Medicine, 2015, 21(6):619-627.
doi: 10.1038/nm.3862
|
|
|
| [50] |
McArthur K, Feng B, Wu Y, et al. MicroRNA-200b regulates vascular endothelial growth factor-mediated alterations in diabetic retinopathy. Diabetes, 2011, 60(4):1314-1323.
doi: 10.2337/db10-1557
pmid: 21357793
|
|
|
| [51] |
Wang B, Herman-Edelstein M, Koh P, et al. E-cadherin expression is regulated by miR-192/215 by a mechanism that is independent of the profibrotic effects of transforming growth factor-beta. Diabetes, 2010, 59(7):1794-1802.
doi: 10.2337/db09-1736
pmid: 20393144
|
|
|
| [52] |
Ślusarz A, Pulakat L. The two faces of miR-29. Journal of Cardiovascular Medicine, 2015, 16(7):480-490.
doi: 10.2459/JCM.0000000000000246
pmid: 25689084
|
|
|
| [53] |
Agarwal V, Bell G W, Nam J W, et al. Predicting effective microRNA target sites in mammalian mRNAs. eLife, 2015, 4:e05005. DOI: 10.7554/eLife.05005.
doi: 10.7554/eLife.05005
|
|
|
| [54] |
Eulalio A, Mano M, Ferro M D, et al. Functional screening identifies miRNAs inducing cardiac regeneration. Nature, 2012, 492(7429):376-381.
doi: 10.1038/nature11739
|
|
|
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