MIT scientists discovered new materials that can support an improved way of delivering messenger RNA into human cells.
This mRNA is a large nucleic acid with the ability to encode genetic information, and can as well direct cells for producing specific proteins. It is not permanently inserted into the genome of a cell unlike DNA, which makes way to use it to produce a therapeutic protein that’s required temporarily. It can be used for producing gene-editing proteins too, altering genomes, and then it will just disappear minimizing the risk of off-target effects.
The large size of mRNA has been challenging for researchers to design it for inserting into cells, and into specific body organs, efficiently. Amino-polyesters that can package mRNA into polymers take care of these issues.
While conducting tests on mice, it was observed that several particles can efficiently deliver mRNA, and can too induce cells for protein synthesizing that’s encoded by mRNA. The best part is that these nanoparticles actually targeted specific organs like the heart, lungs, liver, and spleen. This opens space for researchers to provide specific therapies for certain organs and locations of the human body.
One of the studies they are to perform ahead is what makes these nanoparticles target certain organs. The findings are to be useful in diagnosis and cure of many diseases like pulmonary hypertension, vaccines to immune cells in the spleen, and more. These will be useful even in the CRISPR-Cas9 applications that can handle permanent additions or deletions to a genome!
This research was funded by the Progetto Roberto Roca and the US Defense Advanced Research Projects Agency. Present works of the MIT scientists concern collaboration with the Polytechnic University of Milan on the next-gen of the polymers to improve RNA delivery and to enhance the ability of the particles in targeting specific organs.