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Health: microRNA therapy treats acute myeloid leukemia in mice

Nature Communications

April 27, 2016

Nanoparticles carrying miR-22 - a subset of small, non-coding RNA molecules (microRNA) that regulate gene expression - show therapeutic potential in mouse models of acute myeloid leukemia (AML), reports a study published in Nature Communications this week.

AML is a form of cancer of the blood cells which, despite intensive chemotherapy, is often fatal within five years. Although a lot of information is available regarding the genomic alterations that characterize AML, little is known about the molecular mechanisms that drive transformation of progenitor cells into cancer cells.

Jianjun Chen and colleagues examine tumour samples from 62 human patients and confirm that miR-22 expression is reduced in AML. Using multiple mouse models of AML, they report that restoring miR-22 expression impairs both development and maintenance of AML tumours by suppressing specific cell signalling pathways. The authors then assess the therapeutic potential of miR-22 by delivering miR-22 oligonucleotides through nanoparticles in two leukemic mouse models, one carrying genetically modified mouse leukemia cells, the other one carrying a human-derived leukemia cell line. In both models, treatment with miR-22 resulted in delayed progression and increased survival time.

The possibility of using nanoparticles carrying miR-22 as therapeutic agents to treat AML requires further testing in mouse models before it reaches clinical trials. In addition, the authors propose that combining this treatment with standard chemotherapy agents could also be effective against AML.

doi: 10.1038/ncomms11452

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