Research ArticleCancer Therapy

FolamiRs: Ligand-targeted, vehicle-free delivery of microRNAs for the treatment of cancer

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Science Translational Medicine  02 Aug 2017:
Vol. 9, Issue 401, eaam9327
DOI: 10.1126/scitranslmed.aam9327

Fixing with folate

MicroRNAs (miRNAs), small noncoding nucleotides that regulate gene expression, are attractive therapeutic targets for cancer. The rapid degradation of miRNA mimics in vivo has spurred the use of protection strategies such as liposomes and backbone modification; however, this can hinder miRNA stability, activity, and uptake efficiency. Here, Orellana et al. showed that vehicle-free miRNA could be targeted to cancer cells that overexpress the folate receptor. MiR-34a that was attached to folate, the ligand of the folate receptor, increased miR-34a copy number and reduced tumor size when delivered to mice with lung and breast cancer tumors. Folate-siRNA could also be delivered, suggesting that this conjugation approach for targeted delivery may be compatible with other small RNAs.

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