The Potential for MicroRNA Therapeutics and Clinical Research

Overview

Journal Front Genet

Date 2019 Jun 4

PMID 31156715

Citations 389

Authors

Johora Hanna

Gazi S Hossain

Jannet Kocerha

Affiliations

Soon will be listed here.

Abstract

As FDA-approved small RNA drugs start to enter clinical medicine, ongoing studies for the microRNA (miRNA) class of small RNAs expand its preclinical and clinical research applications. A growing number of reports suggest a significant utility of miRNAs as biomarkers for pathogenic conditions, modulators of drug resistance, and/or as drugs for medical intervention in almost all human health conditions. The pleiotropic nature of this class of nonprotein-coding RNAs makes them particularly attractive drug targets for diseases with a multifactorial origin and no current effective treatments. As candidate miRNAs begin to proceed toward initiation and completion of potential phase 3 and 4 trials in the future, the landscape of both diagnostic and interventional medicine will arguably continue to evolve. In this mini-review, we discuss miRNA drug discovery development and their current status in clinical trials.

Citing Articles

Serum miR-329-3p as a potential biomarker for poor ovarian response in an in vitro fertilization.

Kim J, Kim H, Lee S, Park E, Choi K, Kang K Clin Exp Reprod Med. 2025; 52(1):44-55.

PMID: 40018792 PMC: 11900665. DOI: 10.5653/cerm.2024.07094.

Potential Common Molecular Mechanisms Between Periodontitis and Prostate Cancer: A Network Analysis of Differentially Expressed miRNAs.

Kanli A, Sunnetci-Akkoyunlu D, Kulcu-Sarikaya N, Ugurtas C, Akpinar G, Kasap M In Vivo. 2025; 39(2):795-809.

PMID: 40010958 PMC: 11884493. DOI: 10.21873/invivo.13863.

Preclinical testing of antiviral siRNA therapeutics delivered in lipid nanoparticles in animal models - a comprehensive review.

Idres Y, Idris A, Gao W Drug Deliv Transl Res. 2025; .

PMID: 40000558 DOI: 10.1007/s13346-025-01815-x.

Psoriasis Treatments: Emerging Roles and Future Prospects of MicroRNAs.

Teo L, Juantuah-Kusi N, Subramanian G, Sampath P Noncoding RNA. 2025; 11(1).

PMID: 39997616 PMC: 11858470. DOI: 10.3390/ncrna11010016.

Perspectives in MicroRNA Therapeutics for Cystic Fibrosis.

Finotti A, Gambari R Noncoding RNA. 2025; 11(1.

PMID: 39846681 PMC: 11755495. DOI: 10.3390/ncrna11010003.

References

Lander E, Linton L, Birren B, Nusbaum C, Zody M, Baldwin J . Initial sequencing and analysis of the human genome. Nature. 2001; 409(6822):860-921. DOI: 10.1038/35057062. View

Kola I, Landis J . Can the pharmaceutical industry reduce attrition rates?. Nat Rev Drug Discov. 2004; 3(8):711-5. DOI: 10.1038/nrd1470. View

. Finishing the euchromatic sequence of the human genome. Nature. 2004; 431(7011):931-45. DOI: 10.1038/nature03001. View

Choi C . ABC transporters as multidrug resistance mechanisms and the development of chemosensitizers for their reversal. Cancer Cell Int. 2005; 5:30. PMC: 1277830. DOI: 10.1186/1475-2867-5-30. View

Hopkins A . Network pharmacology: the next paradigm in drug discovery. Nat Chem Biol. 2008; 4(11):682-90. DOI: 10.1038/nchembio.118. View

Mercatelli N, Coppola V, Bonci D, Miele F, Costantini A, Guadagnoli M . The inhibition of the highly expressed miR-221 and miR-222 impairs the growth of prostate carcinoma xenografts in mice. PLoS One. 2008; 3(12):e4029. PMC: 2603596. DOI: 10.1371/journal.pone.0004029. View

Bartel D . MicroRNAs: target recognition and regulatory functions. Cell. 2009; 136(2):215-33. PMC: 3794896. DOI: 10.1016/j.cell.2009.01.002. View

Lanford R, Hildebrandt-Eriksen E, Petri A, Persson R, Lindow M, Munk M . Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science. 2009; 327(5962):198-201. PMC: 3436126. DOI: 10.1126/science.1178178. View

Weber J, Baxter D, Zhang S, Huang D, Huang K, Lee M . The microRNA spectrum in 12 body fluids. Clin Chem. 2010; 56(11):1733-41. PMC: 4846276. DOI: 10.1373/clinchem.2010.147405. View

10.

Weir D, Sturrock A, Leavitt B . Development of biomarkers for Huntington's disease. Lancet Neurol. 2011; 10(6):573-90. DOI: 10.1016/S1474-4422(11)70070-9. View

11.

Pammolli F, Magazzini L, Riccaboni M . The productivity crisis in pharmaceutical R&D. Nat Rev Drug Discov. 2011; 10(6):428-38. DOI: 10.1038/nrd3405. View

12.

Lekprasert P, Mayhew M, Ohler U . Assessing the utility of thermodynamic features for microRNA target prediction under relaxed seed and no conservation requirements. PLoS One. 2011; 6(6):e20622. PMC: 3108951. DOI: 10.1371/journal.pone.0020622. View

13.

Wang R, Li N, Zhang Y, Ran Y, Pu J . Circulating microRNAs are promising novel biomarkers of acute myocardial infarction. Intern Med. 2011; 50(17):1789-95. DOI: 10.2169/internalmedicine.50.5129. View

14.

Kocerha J, Kouri N, Baker M, Finch N, DeJesus-Hernandez M, Gonzalez J . Altered microRNA expression in frontotemporal lobar degeneration with TDP-43 pathology caused by progranulin mutations. BMC Genomics. 2011; 12:527. PMC: 3229715. DOI: 10.1186/1471-2164-12-527. View

15.

Gallo A, Tandon M, Alevizos I, Illei G . The majority of microRNAs detectable in serum and saliva is concentrated in exosomes. PLoS One. 2012; 7(3):e30679. PMC: 3302865. DOI: 10.1371/journal.pone.0030679. View

16.

Washietl S, Will S, Hendrix D, Goff L, Rinn J, Berger B . Computational analysis of noncoding RNAs. Wiley Interdiscip Rev RNA. 2012; 3(6):759-78. PMC: 3472101. DOI: 10.1002/wrna.1134. View

17.

Elfimova N, Schlattjan M, Sowa J, Dienes H, Canbay A, Odenthal M . Circulating microRNAs: promising candidates serving as novel biomarkers of acute hepatitis. Front Physiol. 2012; 3:476. PMC: 3527896. DOI: 10.3389/fphys.2012.00476. View

18.

Janssen H, Reesink H, Lawitz E, Zeuzem S, Rodriguez-Torres M, Patel K . Treatment of HCV infection by targeting microRNA. N Engl J Med. 2013; 368(18):1685-94. DOI: 10.1056/NEJMoa1209026. View

19.

Li Y, Xu M, Gao Z, Wang Y, Yue Z, Zhang Y . Alterations of serum levels of BDNF-related miRNAs in patients with depression. PLoS One. 2013; 8(5):e63648. PMC: 3660391. DOI: 10.1371/journal.pone.0063648. View

20.

Recchioni R, Marcheselli F, Olivieri F, Ricci S, Procopio A, Antonicelli R . Conventional and novel diagnostic biomarkers of acute myocardial infarction: a promising role for circulating microRNAs. Biomarkers. 2013; 18(7):547-58. DOI: 10.3109/1354750X.2013.833294. View