Scientists from Ohio state University have created lipid-based nanoparticles that can deliver genetic material to cells to fight diseases associated with mutations.

Effective and safe delivery of "therapeutic genes" to the body is one of the most important problems in the development of gene therapy. Most often, specially designed viruses are used for this purpose. In the new work, the authors applied lipid-based nanoparticles, which were" Packed " with matrix (informational) RNA molecules — genetic instructions for protein synthesis.

In the body, information RNA is built on the basis of a gene, and then delivered from the cell nucleus to the cytoplasm, where it enters the ribosome — a cellular organ in which a protein is synthesized based on the instructions written in the RNA. But in genetic diseases, mutations in the genes lead to errors in the instructions, so the ribosomes either can not build the right protein molecule at all, or create defective molecules that are not able to perform their functions. In such a situation, the solution may be the introduction of ready-made RNA molecules into the cell with the correct instructions.

In one of the experiments we used genetically modified mice with hemophilia. The nanoparticles were supposed to deliver them information RNA linked to a protein called clotting factor VIII. It is the lack of this protein that is associated with the most frequent hemophilia A (about 80% of all cases of hemophilia). The experiment showed that within 12 hours after the introduction of therapeutic nanoparticles, the mice produced enough clotting factor VIII to provide 90% of its normal activity. The second experiment involved the PCSK9 gene, which is expressed in the liver. There are many known mutations that abnormally increase the activity of this gene, which causes an increase in cholesterol levels in the blood. In this case, the RNA molecule was used as a sample for editing DNA to change the PCSK9 gene, reducing its activity.

The article is published in the journal Science Advances.

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