Polish scientists have made a breakthrough discovery that will help in the treatment of rare genetic diseases.
A team of researchers from the University of Warsaw's Center for New Technologies, the Medical University of Warsaw, and the International Institute of Molecular and Cell Biology have made a groundbreaking discovery. For the first time in the world, they have successfully obtained circular mRNA using a chemical method. This innovative method extends the shelf life of the RNA molecule, thus increasing the production time of therapeutic proteins. In the future, this technology could prove crucial in the treatment of rare genetic diseases such as cystic fibrosis.
Circular RNA is a specific form of messenger RNA that occurs naturally in organisms, but its therapeutic applications are still being explored. The circular RNA structure lacks the ends that are most susceptible to degradation in classic mRNA. This allows circular mRNA to remain in cells much longer, enabling longer and more intensive protein production.
"What we did was, for the first time, encapsulate giant and highly sensitive molecules like RNA using chemical methods in a circular fashion. This allowed us to introduce a structure responsible for translation just as it happens in humans," explains Prof. Jacek Jemielity from the University of Warsaw's Center for New Technologies.
The researchers used a mild chemical reaction that doesn't damage the delicate RNA molecules. Initially, they experimented with short fragments, but surprisingly, the method proved effective even with large molecules up to 4,000 nucleotides long—including those encoding the COVID-19 vaccine. The process yielded up to 60 percent efficiency.
Using a circular form of mRNA allowed for up to 370 times greater protein production than traditional linear mRNA. This opens up new perspectives for therapies that require sustained protein delivery, such as the treatment of genetic diseases.
- These are rare genetic diseases, i.e. diseases in which the therapeutic protein must be delivered much longer than in vaccine applications, because the therapeutic protein must be produced throughout the patient's life - emphasizes Prof. Jemielity.
Cystic fibrosis is an example, where a mutation in a single component of an ion transport protein leads to its dysfunction. Circular mRNA technology could enable the delivery of a correct version of this protein over a long period of time.
Research on this technology is in the early stages. The team plans to increase the process efficiency beyond 80 percent and optimize the RNA structure to make protein production even longer and more efficient. Initial tests in mice have shown that circular RNA is biologically active.
- The final stage will, of course, be demonstrating the applications of circular RNA, meaning we are already thinking about which disease model we want to use to demonstrate that this technology works better than what already exists on the market - announces Prof. Jemielity.
source: Newseria prepared by ZŚ
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