Nanoparticles make targeted gene therapy possible

5th September 2017
Posted By : Enaie Azambuja
Nanoparticles make targeted gene therapy possible

Scientists at the Fred Hutchinson Cancer Research Center in Seattle, Washington have developed a nanoparticle messenger RNA (mRNA) delivery system to temporarily change gene expression in treated cells. Growing specific cell types in a lab and then administering them to patients is the goal of cell therapies, and these types of treatments are coming closer to reality for a variety of diseases.

One way to boost the therapeutic potential of cells is to modify their gene expression. Scientists are still a little wary of permanently modifying gene expression in cells for cell therapy, in case there are unforeseen side effects in patients down the road, such as cancer.

One solution is to deliver mRNA to cells, to temporarily change gene expression. mRNA normally acts as a messenger between our DNA and the machinery that creates new proteins in our cells.

It can increase the expression of specific proteins temporarily, without permanently changing the genes that normally control protein synthesis. However, mRNA tends to be quite delicate and breaks down in the cell very quickly, limiting its usefulness.

The Hutchinson research team came up with a solution consisting of mRNA-loaded nanoparticles. The nanoparticles can be freeze-dried and stored, and when scientists want to use them, they can mix them with water and add them to cells grown in the lab.

Once the nanoparticles bind to the cell surface they are taken inside the cell and cause temporary changes in protein expression. The nanoparticles protect the delicate mRNA in the cell and help it to be effective for a longer time period.

The team tested the technique in several cell types. For example, they used the nanoparticles in T cells to make them more aggressive and effective at destroying cancer cells. The team is now looking to move the technology into clinical trials and hopes to try it in humans soon.


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