New delivery method for microRNA cancer treatment

Short strands of RNA known as microRNA help cells to fine-tune their gene expression. Disruption or loss of some microRNAs has been linked to cancer, raising the possibility of treating tumors by adjusting microRNA levels.

Developing such treatments requires delivering microRNA to tumors, which has proven difficult. However, researchers from MIT have now shown that by twisting RNA strands into a triple helix and embedding them in a biocompatible gel, they can not only deliver the strands efficiently but also use them to shrink aggressive tumors in mice.

Using this technique, the researchers dramatically improved cancer survival rates by simultaneously turning on a tumor-suppressing microRNA and de-activating one that causes cancer. They believe their approach could also be used for delivering other types of RNA, as well as DNA and other therapeutic molecules.

“This is a platform that can deliver any gene of interest,” says Natalie Artzi, a research scientist in MIT’s Institute for Medical Engineering and Science (IMES) and an assistant professor of medicine at Brigham and Women’s Hospital. “This work demonstrates the promise of local delivery in combating cancer. In particular, as relates to gene therapy, the triplex structure improves RNA stability, uptake, and transfection efficacy.”

Characterization of RNA-dendrimer nanoconjugates. (a-d) High-resolution SEM images of the 3D structure of the RNA triple-helix formations. The samples were coated with gold (10-15 nm thickness). Micron level RNA aggregates (a-c) are formed by interactions between the RNA triplex nanoparticles (d).

Nature Materials – Self-assembled RNA-triple-helix hydrogel scaffold for microRNA modulation in the tumor microenvironment

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