IOCB Prague is expanding the possibilities of using RNA in gene medicine

15. 05. 2024

Dr Petr Cígler and his collaborators are working on refining molecular systems for transporting ribonucleic acid (RNA) molecules into cells. The question of how to effectively deliver RNA to a designated place in the body in order to silence a malfunctioning gene is one of the greatest challenges of the rapidly developing field of gene medicine. Now, researchers from IOCB Prague have taken yet another significant step towards achieving this goal. They have described in detail the preparation of a novel composite vector nanomaterial for transporting RNA, in which they focused mainly on ensuring its non-toxicity to cells. The reason is that the harmfulness of hitherto known nucleic acid vectors presents a serious obstacle to the expansion of gene therapy. A new article on the topic has now been published in the scientific journal Advanced Functional Materials.

Petr Cígler and his colleagues worked with the assumption that the toxicity of nucleic acid vectors is caused by an excessive accumulation of positive charges along the chains of carrier polymers that bind nucleic acids. Therefore, they systematically diluted this positive charge with uncharged monomers and found that its slight dilution did not impair the ability of the system to transport nucleic acids. In their thorough study, they also describe the circumstances under which new materials can be prepared for use in gene medicine. The researchers paid close attention to the conditions in which these substances bind nucleic acids most effectively and in what stoichiometric proportion.

Cells can regulate the translation of genes from mRNA to proteins in several ways. One of them is to silence a gene with the help of an siRNA (small interfering RNA) molecule. This blocks the production of the target protein, which is especially useful if the gene encoding is mutated or otherwise defective. In addition, it is possible to intervene if the innate mechanisms of translation regulation are impaired. In such a case, too much of a protein is produced in the body, which is detrimental to its health.

‘Gene therapy is not only highly effective, but also acutely targeted. Its biggest advantage lies in the fact that it accurately targets individual mutations or disorders without the danger of missing the mark. Using siRNA, it is possible to hit even a single mutated “letter” of a gene,’ says Marek Kindermann, the first author of the study, listing the main advantages of gene therapy. Currently, five drugs based on siRNA are approved for worldwide use, and there are about a dozen more on the ‘waiting list’.

Read the full article on the IOCB's website.

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