Jerusalem, 17 September, 2025 (TPS-IL) — Israeli scientists have developed a new gene therapy method that could transform the treatment of inflammatory bowel diseases, including Crohn’s disease and ulcerative colitis, Tel Aviv University announced on Wednesday.
Using advanced RNA molecules called locked nucleic acids (LNAs), the research team achieved strong anti-inflammatory effects in mice without any side effects.
Globally, an estimated 6.8 million people are living with inflammatory bowel disease (IBD), which includes Crohn’s disease and ulcerative colitis.
The breakthrough relies on tiny fat-based carriers called lipid nanoparticles, which deliver the LNAs directly to the affected tissue. This targeted approach allows for much lower doses than previously required, making treatment safer and potentially more affordable.
The findings were published the peer-reviewed Nature Communications journal.
“Our study focused on unique RNA molecules called LNA. Unlike most RNA molecules, LNA molecules are very stable and do not break down easily. Until about 10 years ago, they were thought to have great potential as genetic drugs. But previous experiments showed that very high doses were needed, which caused severe side effects and made treatment costly. We wanted to find a better, more precise approach,” said Prof. Dan Peer, a leading expert in RNA therapeutics and nanomedicine.
The research team, including Neubauer doctoral student Shahd Qassem and postdoctoral fellow Dr. Gonna Somu Naidu, tested the method in mice with chronic bowel inflammation. They used an LNA molecule that silences the TNFα gene, known to drive inflammation in IBD. By screening a library of lipids developed over 13 years in Prof. Peer’s lab, they found the optimal carrier for the LNA.
The results were striking. The treatment worked at a dose 30 times lower than previous LNA therapies, and no side effects were observed. “At this lower dose, delivered precisely to the correct site, the drug proved highly effective in treating the disease without causing any side effects,” Peer said.
This method could make gene therapy for IBD safer, more effective, and more practical for patients. Current treatments often have limited effectiveness or cause significant side effects, so a targeted therapy could significantly improve quality of life.
Because many rare genetic diseases are caused by a single defective gene, encapsulating RNA molecules like LNAs in lipid nanoparticles could silence or correct faulty genes, opening up therapies for conditions that currently have no treatment.
“Our study paves the way to developing new LNA-based drugs for inflammatory bowel diseases, as well as a wide range of other diseases – including rare genetic disorders, vascular and heart diseases, and neurological diseases such as Parkinson’s and Huntington’s,” Peer said.
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