Small molecules targeting microRNA for cancer therapy: Promises and obstacles

Di Wen, Michael Danquah, Amit Kumar Chaudhary, Ram I. Mahato

Research output: Contribution to journalReview articlepeer-review

82 Scopus citations


Aberrant expression of miRNAs is critically implicated in cancer initiation and progression. Therapeutic approaches focused on regulating miRNAs are therefore a promising approach for treating cancer. Antisense oligonucleotides, miRNA sponges, and CRISPR/Cas9 genome editing systems are being investigated as tools for regulating miRNAs. Despite the accruing insights in the use of these tools, delivery concerns have mitigated clinical application of such systems. In contrast, little attention has been given to the potential of small molecules to modulate miRNA expression for cancer therapy. In these years, many researches proved that small molecules targeting cancer-related miRNAs might have greater potential for cancer treatment. Small molecules targeting cancer related miRNAs showed significantly promising results in different cancer models. However, there are still several obstacles hindering the progress and clinical application in this area. This review discusses the development, mechanisms and application of small molecules for modulating oncogenic miRNAs (oncomiRs). Attention has also been given to screening technologies and perspectives aimed to facilitate clinical translation for small molecule-based miRNA therapeutics.

Original languageEnglish (US)
Pages (from-to)237-247
Number of pages11
JournalJournal of Controlled Release
StatePublished - Dec 10 2015


  • Cancer therapy
  • OncomiR
  • Small molecule miRNA inhibitors
  • miRNA

ASJC Scopus subject areas

  • Pharmaceutical Science


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