Researchers at Karolinska Institutet have developed a technique that enables efficient delivery of therapeutic proteins and RNA to cells. The method, published in Nature Communications, shows promising results in animal studies to deliver gene editors and protein therapeutics.
The method is based on so-called extracellular vesicles (EVs), tiny bubbles that are naturally secreted by cells and can transport biologically active molecules between cells. Researchers have now succeeded in improving these bubbles by introducing two key components: a small part of a bacterial protein called intein and a so-called fusogenic protein from a virus.
The fusogenic protein helps the bubbles to fuse with the endosomal membrane and release their contents into the cell, while the intein can cut itself and thus help release therapeutic proteins inside the cell.
“This innovative engineering strategy represents a major step forward for extracellular vesicle technology, effectively overcoming key barriers such as poor endosomal escape and limited intracellular release,” says Professor Samir EL Andaloussi, last author of the study and researcher at the Department of Laboratory Medicine.
“Our in vivo findings highlight the potential of engineered EVs as a versatile platform for delivering therapeutics to treat a broad range of conditions, including systemic inflammation, genetic diseases, and neurological disorders.”
The study’s first author, Dr. Xiuming Liang at the Department of Laboratory Medicine, adds, “By improving the efficiency and reliability of therapeutic delivery into target cells, this technology could significantly broaden the application of advanced medicines.”
Brain changes in mice
In experiments on cells and live animals, the researchers were able to efficiently deliver Cre recombinase, a protein that can cut and paste DNA, and Cas9/sgRNA complexes, which are used to edit genes. When extracellular vesicles loaded with Cre recombinase were injected into the brains of mice, a significant change in cells in the hippocampus and cortex brain structures was observed.
“This gives hope to use the CRISPR/Cas9 gene scissors or similar tools to treat severe genetic diseases of the central nervous system, such as Huntington’s disease and spinal muscular atrophy,” says Liang.
The researchers also demonstrated that the technique could be used to treat systemic inflammation in mice.
More information: Xiuming Liang et al, Engineering of extracellular vesicles for efficient intracellular delivery of multimodal therapeutics including genome editors, Nature Communications (2025). DOI: 10.1038/s41467-025-59377-y. www.nature.com/articles/s41467-025-59377-y
Journal information: Nature Communications
News
Smaller Than a Grain of Salt: Engineers Create the World’s Tiniest Wireless Brain Implant
A salt-grain-sized neural implant can record and transmit brain activity wirelessly for extended periods. Researchers at Cornell University, working with collaborators, have created an extremely small neural implant that can sit on a grain of [...]
Scientists Develop a New Way To See Inside the Human Body Using 3D Color Imaging
A newly developed imaging method blends ultrasound and photoacoustics to capture both tissue structure and blood-vessel function in 3D. By blending two powerful imaging methods, researchers from Caltech and USC have developed a new way to [...]
Brain waves could help paralyzed patients move again
People with spinal cord injuries often lose the ability to move their arms or legs. In many cases, the nerves in the limbs remain healthy, and the brain continues to function normally. The loss of [...]
Scientists Discover a New “Cleanup Hub” Inside the Human Brain
A newly identified lymphatic drainage pathway along the middle meningeal artery reveals how the human brain clears waste. How does the brain clear away waste? This task is handled by the brain’s lymphatic drainage [...]
New Drug Slashes Dangerous Blood Fats by Nearly 40% in First Human Trial
Scientists have found a way to fine-tune a central fat-control pathway in the liver, reducing harmful blood triglycerides while preserving beneficial cholesterol functions. When we eat, the body turns surplus calories into molecules called [...]
A Simple Brain Scan May Help Restore Movement After Paralysis
A brain cap and smart algorithms may one day help paralyzed patients turn thought into movement—no surgery required. People with spinal cord injuries often experience partial or complete loss of movement in their arms [...]
Plant Discovery Could Transform How Medicines Are Made
Scientists have uncovered an unexpected way plants make powerful chemicals, revealing hidden biological connections that could transform how medicines are discovered and produced. Plants produce protective chemicals called alkaloids as part of their natural [...]
Scientists Develop IV Therapy That Repairs the Brain After Stroke
New nanomaterial passes the blood-brain barrier to reduce damaging inflammation after the most common form of stroke. When someone experiences a stroke, doctors must quickly restore blood flow to the brain to prevent death. [...]
Analyzing Darwin’s specimens without opening 200-year-old jars
Scientists have successfully analyzed Charles Darwin's original specimens from his HMS Beagle voyage (1831 to 1836) to the Galapagos Islands. Remarkably, the specimens have been analyzed without opening their 200-year-old preservation jars. Examining 46 [...]
Scientists discover natural ‘brake’ that could stop harmful inflammation
Researchers at University College London (UCL) have uncovered a key mechanism that helps the body switch off inflammation—a breakthrough that could lead to new treatments for chronic diseases affecting millions worldwide. Inflammation is the [...]
A Forgotten Molecule Could Revive Failing Antifungal Drugs and Save Millions of Lives
Scientists have uncovered a way to make existing antifungal drugs work again against deadly, drug-resistant fungi. Fungal infections claim millions of lives worldwide each year, and current medical treatments are failing to keep pace. [...]
Scientists Trap Thyme’s Healing Power in Tiny Capsules
A new micro-encapsulation breakthrough could turn thyme’s powerful health benefits into safer, smarter nanodoses. Thyme extract is often praised for its wide range of health benefits, giving it a reputation as a natural medicinal [...]
Scientists Develop Spray-On Powder That Instantly Seals Life-Threatening Wounds
KAIST scientists have created a fast-acting, stable powder hemostat that stops bleeding in one second and could significantly improve survival in combat and emergency medicine. Severe blood loss remains the primary cause of death from [...]
Oceans Are Struggling To Absorb Carbon As Microplastics Flood Their Waters
New research points to an unexpected way plastic pollution may be influencing Earth’s climate system. A recent study suggests that microscopic plastic pollution is reducing the ocean’s capacity to take in carbon dioxide, a [...]
Molecular Manufacturing: The Future of Nanomedicine – New book from Frank Boehm
This book explores the revolutionary potential of atomically precise manufacturing technologies to transform global healthcare, as well as practically every other sector across society. This forward-thinking volume examines how envisaged Factory@Home systems might enable the cost-effective [...]
New Book! NanoMedical Brain/Cloud Interface – Explorations and Implications
New book from Frank Boehm, NanoappsMedical Inc Founder: This book explores the future hypothetical possibility that the cerebral cortex of the human brain might be seamlessly, safely, and securely connected with the Cloud via [...]
