Expert Review

Pharmaceutical Research

, Volume 28, Issue 12, pp 3030-3042

First online:

microRNA Replacement Therapy for Cancer

  • Jon C. HenryAffiliated withCollege of Pharmacy, Ohio State UniversityDepartment of Surgery, Ohio State University
  • , Ana Clara P. Azevedo-PoulyAffiliated withCollege of Pharmacy, Ohio State University
  • , Thomas D. SchmittgenAffiliated withCollege of Pharmacy, Ohio State University Email author 

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microRNA are small noncoding RNAs that translationally repress their target messenger RNAs. Many microRNAs are expressed at reduced levels in tumors. microRNAs with reduced expression in cancer often regulate oncogenes, resulting in enhanced tumor growth. One therapeutic option is to restore microRNA levels in the tumor to that of the non-diseased tissue. This is possible by delivering microRNA to the tumor in the form of an oligonucleotide mimic or by expressing the microRNA in the cancer using a gene vector. This article surveys the field of oligonucleotide mimics and gene vector approaches to restore microRNA levels in tumors and reviews the literature on experimental and pre-clinical studies that have used these approaches to treat cancer.


microRNA mimetic microRNA mimic therapeutic microRNAs cancer treatment