Researchers at Karolinska Institutet have discovered an innovative method. This innovative approach represents a new paradigm that greatly improves the ability to deliver therapeutic proteins and RNA to cells. Dr. Xiuming Liang, of the Department of Laboratory Medicine, heads up this cutting-edge inquiry. It opens the door to deliver innovative medicines in a more efficient manner. The findings were published in the esteemed journal Nature Communications and can be accessed through DOI: 10.1038/s41467-025-59377-y.
The key to the approach is engineering extracellular vesicles, which are small membrane-bound structures that help cells communicate with one another. By improving these vesicles, researchers have developed an effective technology for intracellular delivery of multimodal therapeutics to make a significant impact on cancer therapy. This study added evidence that engineered vesicles offer the potential for safe, effective delivery of therapeutic proteins and RNA. This groundbreaking discovery turns them into invaluable resources for future medicinal applications.
Engineering Extracellular Vesicles for Therapeutic Applications
Dr. Liang and her colleagues focused on improving the functionality of extracellular vesicles by incorporating two pivotal components: a small part of a bacterial protein known as intein and a fusogenic protein derived from a virus. These improvements further augment the vesicles’ therapeutic payload delivery capacity and complexity as enabled multi-therapeutic agents are delivered into target cells.
The research documents the development of two innovative systems — VEDIC and VFIC. These systems are tailored to achieve efficient intracellular delivery of proteins, including effects observed in-vitro and in-vivo. This progress is very important. It enables us to target and deliver therapeutic proteins and RNA with high precision, helping us establish optimal, effective treatment strategies for diseases ranging from cancer to cardiovascular disease.
Professor Samir El Andaloussi, principal investigator at the Department of Laboratory Medicine, spoke about the promise of these custom-made EVs in medicine. As one of the study’s last authors, he’s highly experienced in this area, which is probably why… The ability to deliver genome editors and other therapeutic agents effectively could lead to significant breakthroughs in treating genetic disorders and other health conditions.
Implications for Future Medical Research
The application of this research goes well beyond pure science. The technique’s unique ability to allow for high level medicine that is otherwise not possible to deliver creates incredible new opportunities for therapeutic interventions. With the potential to apply these engineered vesicles in various medical fields, researchers are optimistic about their impact on patient care.
The scientific community, meanwhile, is hard at work looking to unlock the versatility of extracellular vesicles. This study provides a strong evidence base moving forward to create safer and more efficacious treatment alternatives. By enabling spatially precise and temporally regulated delivery of therapeutics, LEAP can enhance the efficacy of existing therapies. Beyond that, it opens more possibilities for novel treatments that would otherwise be too challenging to deliver.
This novel strategy could supercharge current gene therapy methods. This allows a more precise targeted intervention with diseases at the molecular level. As knowledge and research moves forward, we will have to study more. This will allow us to better understand the range of applications allowed and to pinpoint any limitations.