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Simulations show how to make gene therapy more effective Apr 22, 2020 | 13:35 / Interesting information

Diseases with a genetic cause could, in theory, be treated by supplying a correct version of the faulty gene. However, in practice, delivering new genetic material to human cells is difficult. Scientists at the University of Groningen consider the method for the delivery of such genes by DNA/lipid complexes (lipoplexes) as promising. The idea behind gene therapy is very simple: if a disease is caused by a particular version of a single gene, it could be cured by replacing this gene. However, our bodies are very good at destroying foreign DNA, so delivering a new gene inside a cell is difficult. Viruses are very good at delivering genetic material into cells, but they can also trigger the body's defenses, creating an immune response that can make the patient sick and that in rare cases has even proved lethal. That is why scientists are now experimenting with lipid-based complexes, which are similar to cell membranes. These lipoplexes are taken up by cells in a structure called an endosome. The problem is that endosomes digest material, so the DNA needs to escape quickly. The lipoplex can fuse with the endosome membrane, allowing the DNA to enter the cell. This should happen as efficiently as possible to prevent degradation. Therefore, the interaction between the lipoplex and the endosome membrane is on the focus of scientists. Studying this interaction through experiments is difficult. That is why the scientists decided to simulate the interaction. They predict that poly-unsaturated fatty acids will speed up the fusion, allowing the DNA to escape into the cell's cytoplasm more quickly. This prediction could now be tested in the lab.

https://www.sciencedaily.com