TY - JOUR
T1 - Strategies to modulate microRNA functions for the treatment of cancer or organ injury
AU - Lee, Tae Jin
AU - Yuan, Xiaoyi
AU - Kerr, Keith
AU - Yoo, Ji Young
AU - Kim, Dong H.
AU - Kaur, Balveen
AU - Eltzschig, Holger K.
N1 - Funding Information:
This work was supported in part by the National Institutes of Health (NIH) National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) [Grants R01-DK097075 and R01-DK109574] and the NIH National Heart, Lung, and Blood Institute (NHLBI) [Grants P0I-HL114457, R01-HL109233, R01-HL119837, and R01-HL133900] to H.K.E.; Pilot/Feasibility Program from Texas Medical Center Digestive Diseases Center through NIH NIDDK [Grant P30 DK056338] to T.J.L.; the American Thoracic Society Unrestricted Grant, American Heart Association Career Development Award [19CDA34660279], American Lung Association Catalyst Award [CA-622265], and The Center for Clinical and Translational Sciences, McGovern Medical School Pilot Award [1UL1TR003167–01], to X.Y.; American Cancer Society Research Scholar Grants [RSG-19-185-01-MPC] to J.Y.Y.; NIH National Cancer Institute (NCI) [Grant R01 CA150153 and P01 CA163205] and NIH National Institute of Neurologic Disorders and Stroke (NINDS) [Grant R01 NS064607] to B.K.; and NIH NINDS [Grant R01 NS104280] to D.H.K. https://doi.org/10.1124/pr.119.019026.
Publisher Copyright:
© 2020 by The American Society for Pharmacology and Experimental Therapeutics.
PY - 2020/7
Y1 - 2020/7
N2 - Cancer and organ injury—such as that occurring in the perioperative period, including acute lung injury, myocardial infarction, and acute gut injury —are among the leading causes of death in the United States and impose a significant impact on quality of life. MicroRNAs (miRNAs) have been studied extensively during the last two decades for their role as regulators of gene expression, their translational application as diagnostic markers, and their potential as therapeutic targets for disease treatment. Despite promising preclinical outcomes implicating miRNA targets in disease treatment, only a few miRNAs have reached clinical trials. This likely relates to difficulties in the delivery of miRNA drugs to their targets to achieve efficient inhibition or overexpression. Therefore, understanding how to efficiently deliver miRNAs into diseased tissues and specific cell types in patients is critical. This review summarizes current knowledge on various approaches to deliver therapeutic miRNAs or miRNA inhibitors and highlights current progress in miRNA-based disease therapy that has reached clinical trials. Based on ongoing advances in miRNA delivery, we believe that additional therapeutic approaches to modulate miRNA function will soon enter routine medical treatment of human disease, particularly for cancer or perioperative organ injury. Significance Statement——MicroRNAs have been studied extensively during the last two decades in cancer and organ injury, including acute lung injury, myocardial infarction, and acute gut injury, for their regulation of gene expression, application as diagnostic markers, and therapeutic potentials. In this review, we specifically emphasize the pros and cons of different delivery approaches to modulate microRNAs, as well as the most recent exciting progress in the field of therapeutic targeting of microRNAs for disease treatment in patients.
AB - Cancer and organ injury—such as that occurring in the perioperative period, including acute lung injury, myocardial infarction, and acute gut injury —are among the leading causes of death in the United States and impose a significant impact on quality of life. MicroRNAs (miRNAs) have been studied extensively during the last two decades for their role as regulators of gene expression, their translational application as diagnostic markers, and their potential as therapeutic targets for disease treatment. Despite promising preclinical outcomes implicating miRNA targets in disease treatment, only a few miRNAs have reached clinical trials. This likely relates to difficulties in the delivery of miRNA drugs to their targets to achieve efficient inhibition or overexpression. Therefore, understanding how to efficiently deliver miRNAs into diseased tissues and specific cell types in patients is critical. This review summarizes current knowledge on various approaches to deliver therapeutic miRNAs or miRNA inhibitors and highlights current progress in miRNA-based disease therapy that has reached clinical trials. Based on ongoing advances in miRNA delivery, we believe that additional therapeutic approaches to modulate miRNA function will soon enter routine medical treatment of human disease, particularly for cancer or perioperative organ injury. Significance Statement——MicroRNAs have been studied extensively during the last two decades in cancer and organ injury, including acute lung injury, myocardial infarction, and acute gut injury, for their regulation of gene expression, application as diagnostic markers, and therapeutic potentials. In this review, we specifically emphasize the pros and cons of different delivery approaches to modulate microRNAs, as well as the most recent exciting progress in the field of therapeutic targeting of microRNAs for disease treatment in patients.
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U2 - 10.1124/pr.119.019026
DO - 10.1124/pr.119.019026
M3 - Article
C2 - 32554488
AN - SCOPUS:85086692445
SN - 0031-6997
VL - 72
SP - 639
EP - 667
JO - Pharmacological Reviews
JF - Pharmacological Reviews
IS - 3
ER -