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
Researcher at Russian plague laboratory dies of ‘unknown’ infection
Russian authorities have imposed "anti-epidemic measures" after a laboratory worker at an institute in Siberia dedicated to studying the plague and other infectious diseases died of a mysterious illness. The researcher, a 28-year-old female [...]
GHCE Concept
From the preface of the book Global Health Care Equivalency in the Age of Nanotechnology, Nanomedicine and Artificial Intelligence, Edited by Frank Boehm: Since the publication of my first book (Nanomedical Device and Systems [...]
A Surprising Brain Signal Could Explain Why Eating Less Protein Extends Lifespan
A new paper suggests that limiting protein intake triggers a coordinated response throughout the body that may help explain how protein restriction affects healthy aging. Animals respond to protein restriction, or reduced protein in [...]
Back and shoulder surgery is often worse than useless
Millions of operations should be scrapped SIX IN TEN Britons have surgery at some point. So do lots of Americans, at a cost of perhaps $500bn a year, a third of all spending on [...]
Cocaine plays with mind, hijacks brain circuit, drives increasingly rigid and repetitive behavior
Researchers have found that cocaine hijacks brain circuit, which to drive increasingly rigid and repetitive behavior. The brain circuit that normally participates in selecting natural mouth and face movements, but which becomes disproportionately engaged [...]
Injectable nanodevices could provide effective treatment for drug-resistant glioblastoma
The brain cancer glioblastoma is one of the most aggressive and treatment-resistant cancers known to medicine, carrying a median survival of just 12-15 months, even with the best available care. Now, researchers at the [...]
New Treatment Cuts “Bad” Cholesterol in Half for a Full Year
A single infusion of an experimental CRISPR-Cas9 therapy was reported to safely lower LDL cholesterol by 52.5% and triglycerides by 47.8%, as measured 12 months after treatment. What if one infusion could keep cholesterol [...]
Too Much RNA Can Drain a Cell’s Energy, Scientists Discover
A virus may cripple a cell’s power supply simply by producing too much of a molecule the cell needs to survive. New research from the Texas A&M College of Veterinary Medicine and Biomedical Sciences [...]
West African scientists warn of weakening health systems and urge stronger outbreak detection
LOME, Togo (AP) — West African laboratory scientists called on governments Friday to strengthen capacity to detect disease outbreaks to avoid another major outbreak, saying health systems across the region are becoming weaker. Participants [...]
A Little-Known Protein Could Point to a New Way To Treat Alzheimer’s
A study suggests that increasing SORLA protein levels could help treat Alzheimer’s disease and other disorders involving tau protein. Increasing SORLA, a protein with a protective role in the brain, helped mice withstand damage [...]
Cancer’s Hidden Antioxidant Shield Helps It Escape the Immune System
Blocking an antioxidant protein that tumors use to suppress immune attacks improved cancer immunotherapy responses in mice. Cancer cells can release antioxidants that interfere with the immune cells trying to kill them. Researchers have [...]
Scientists Find a Berry Compound That Helps Muscle Cells Burn Fat
Pterostilbene, a compound found naturally in some foods, affects how skeletal muscles process fats by stabilizing a protein called PPARδ and increasing its signaling activity. Pterostilbene, a natural compound found in blueberries, grapes, and [...]
Two Hidden Forces Help Build the Human Brain Before Birth
Scientists have uncovered two surprising forces that help guide how the human brain forms before birth. Before birth, the human brain is shaped in large part by an unusual class of stem cells known [...]
Researchers Uncover a Hidden Trigger Behind Chronic Inflammation
The findings offer new insights that could help guide the development of future therapies. A protein called human resistin may help flip on one of the immune system’s most powerful inflammatory switches. Researchers at [...]
Scientists Have Uncovered Previously Hidden Microbial Activity on Human Skin
The most abundant microbes on your skin may not be the ones doing most of the work. Human skin supports vast communities of bacteria, fungi, and viruses that can influence its protective barrier, immune [...]
Brazilian Tree Compounds Fight COVID-19 on Multiple Fronts
Scientists found compounds in a Brazilian tree that hit SARS-CoV-2 on multiple fronts, revealing a promising new lead in the search for COVID-19 treatments. Researchers have found that galloylquinic acids extracted from the leaves [...]
