Researchers at the University of Toronto have developed a new, innovative approach to tackling genetic diseases. According to a study published in the journal Science, they have created a next-generation RNA therapy that helps cells correct genetic errors and produce proteins essential for the body’s survival.
The main cause of thousands of rare diseases, including cystic fibrosis and a number of neurological disorders, is a so-called “premature stop signal” in the genetic code. Because of this error, the body stops producing the required protein halfway through the process. As a result, cells lose their function and disease develops. The new therapy, however, forces cells to ignore this signal, allowing them to complete protein production.
Until now, developing a separate gene therapy for each rare mutation has been an extremely difficult, costly and time-consuming process. The key advantage of the new approach developed by Canadian scientists is that it is universal. The therapy could offer a common strategy for treating thousands of different diseases caused by the same genetic error, significantly simplifying the development of treatments.
To achieve these results, the researchers combined the latest advances in chemistry and biology. To safely deliver the new drug to cells, they used a specialized delivery system — lipid nanoparticles. This is the same technology that proved successful in COVID-19 vaccines, although it has now been specifically adapted to the requirements of the new therapy.
The technology has already been tested in laboratory models of cystic fibrosis, a severe genetic disease. The researchers found that combining the new therapy with existing medications produced the best results. The innovative RNA therapy prompts cells to resume production of the missing protein, after which the conventional drug activates that protein.
Experts say the unprecedented finding could take the development of genetic therapies to an entirely new level. Researchers are now working to simplify the way the treatment is delivered. For example, in the case of lung diseases, the technology could eventually allow patients to receive the therapy at home, rather than in a hospital, using a conventional inhaler.
