Scientists at Rutgers University–Newark have developed a first-of-its-kind RNA-based nanotechnology that assembles itself inside living human cells and can be programmed to stop propagation of harmful cells.

The findings, recently accepted for publication in Nature Communications, represent a major breakthrough in biomedical research. The researchers are now in the midst testing the technology on human cancer cells as a potential cure for the disease but have not yet finished the study or published results.

This nanostructure technology, which was tested in human cell cultures, can be used as a molecular tool for biomedical research and therapeutics. Because it can be customized, it has the versatility to target multiple detrimental genes and proteins simultaneously.

The work was led by Professor Fei Zhang of the Rutgers-Newark Department of Chemistry and Professor Jean-Pierre Etchegaray of the Department of Biological Sciences at Rutgers-Newark, along with an interdisciplinary team of researchers. Watch them explain their breakthrough in this video.

“We are providing the method, a new design strategy for artificial RNA structures with programmable functions,” said Zhang.

The technology works because every cell in the body runs on instructions stored in DNA.  RNA– which scientists compare to software–acts as the messenger, carrying instructions that tell the cell what proteins to make.

Instead of delivering pre-built molecules into a cell, the Rutgers–Newark team designed a way to give the cell a synthetic DNA template.

What’s new is that this RNA is designed to fold and assemble itself into precise shapes and controllable localization inside the cell. The assembled RNA structures carry functional domains that can be reprogrammed for different biomedical applications.

These artificial RNA will be generated and assembled inside the cell, rather than synthesized and delivered from outside. That’s one of the critical aspects of this nanotechnology that is new. The RNA pieces behave like tiny Lego blocks that find each other and snap together automatically. They can also be redesigned into different types of geometries with new functions.

That capability is especially important for cancer, which is driven by many malfunctioning genes acting together, said Etchegaray. The approach is designed to affect cancer cells while sparing healthy cells by tailoring the RNA structures to recognize disease-specific signals.

The Rutgers-Newark researchers have started using the technology to see if they can disable cancer stem cells and stop them from multiplying.

“We are trying right now to use this technology to target oncogenes and see if we can disable cancer stem cells, which are considered cancer initiating and propagating cells with therapeutic resistance,’’ Etchegaray said. “They will no longer be able to promote tumor growth, metastasis and even relapse,’’ said Etchegaray.

Most current RNA-based therapies are designed to target one molecule at a time. This new platform can be programmed to interact with multiple targets simultaneously, which marks an unprecedent potential for biotechnological applications, said Zhang and Etchegaray.

The technology can also be adapted to work together and enhance existing RNA therapies. “We can integrate fragments and functional sequences from the traditional RNA therapeutics into our platforms,” Zhang said.

The researchers have an approved provisional patent and are actively seeking investors, industry collaborators, and research partners to help accelerate development, including clinical trials.

“If we can have more people on board and attract different interest from partners, that will make this going forward faster,” said Zhang.

“Apart from cancer, we can customize this nanotechnology to target other diseases driven by misexpression of genes and proteins,” added Etchegaray.