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
Targeting Nanoparticles for Heart Repair
Scientists have engineered dual-membrane nanoparticles that home in on heart tissue after a heart attack, delivering regenerative molecules while evading the body’s immune defences. Myocardial infarction, better known as a heart attack, is a [...]
Natural Compound Combo Restores Aging Brain Cells
Scientists have identified a natural compound combination that reverses aging-related brain cell decline and removes harmful Alzheimer’s-linked proteins. The treatment, combining nicotinamide (vitamin B3) and the green tea antioxidant epigallocatechin gallate, restores guanosine triphosphate [...]
Silver Nanoparticles Get a Green Makeover: An Eco-Friendly Way to Target Diabetes
Researchers have developed an eco-friendly method to produce silver nanoparticles from the roots of Martynia annua, showing strong antioxidant and anti-diabetic potential while avoiding the toxic by-products of conventional synthesis. Silver nanoparticles are particularly popular in research because [...]
Quantum Breakthrough: Scientists Find “Backdoor” to 60-Year-Old Superconducting Mystery
A Copenhagen team has unlocked a clever “backdoor” into studying rare quantum states once thought beyond reach. Scientists at the Niels Bohr Institute, University of Copenhagen, have discovered a new approach for investigating rare [...]
3D-Printed Nylon Filters With Titanium Dioxide For Greywater Treatment
A team of researchers has developed a novel water filtration system that combines nanotechnology with 3D printing, aiming to create a low-cost, sustainable solution for greywater treatment. As reported in Micro & Nano Letters, the study demonstrates this [...]
New COVID variant ‘Stratus’ is spreading in the U.S. and worldwide
A new COVID variant is climbing the ranks in the U.S., becoming the third-most common strain of the summer. Variant XFG, colloquially known as "Stratus," was first detected in Southeast Asia in January but [...]
Fat Molecule May Control How You Feel Emotion
Key Questions Answered Q: What did researchers discover about the serotonin 5-HT1A receptor? A: They mapped how it activates different brain signaling pathways, offering insight into how mood and emotion are regulated at the [...]
Nanodevice uses sound to sculpt light, paving the way for better displays and imaging
Light can behave in very unexpected ways when you squeeze it into small spaces. In a paper in the journal Science, Mark Brongersma, a professor of materials science [...]
ChatGPT helps speed up patient screening for clinical trials
A new study in the academic journal Machine Learning: Health discovers that ChatGPT can accelerate patient screening for clinical trials, showing promise in reducing delays and improving trial success rates. Researchers at UT Southwestern Medical Centre used [...]
New Study Reveals This Popular Fruit Is Actually a “Superfood”
A new peer-reviewed article argues that grapes deserve a place among today’s top superfoods. A recent article published in the peer-reviewed Journal of Agriculture and Food Chemistry takes a closer look at the term [...]
Experimental Drug Reverses PTSD Symptoms in Mice – Already in Human Trials
Excessive levels of GABA released by astrocytes impair the brain’s ability to extinguish fear responses in PTSD, but a newly identified drug target offers promising hope for treatment. Many people with post-traumatic stress disorder (PTSD) [...]
New high-selectivity nanozyme enables rapid and visible disease diagnostics
To enable early diagnosis of acute illnesses and effective management of chronic conditions, point-of-care testing (POCT) technology—diagnostics conducted near the patient—is drawing global attention. The key to POCT lies in enzymes that recognize and [...]
Globally, over 2.5 million COVID deaths prevented worldwide thanks to vaccines
Thanks to vaccinations against SARS-CoV-2 in the period 2020–2024, 2.533 million deaths were prevented at the global level; one death was avoided for every 5,400 doses of vaccine administered. Some 82% of the lives [...]
RNA-seq outperforms DNA methods in detecting actionable cancer mutations
Hospital for Sick Children in Toronto researchers are reporting that targeted RNA sequencing can detect clinically actionable alterations in 87% of tumors and provide decisive findings where DNA-seq either fails, returns no variant, or [...]
Physicists discover new state of quantum matter
Researchers at the University of California, Irvine have discovered a new state of quantum matter. The state exists within a material that the team reports could lead to a new era of self-charging computers [...]
Researchers create safer nonstick surface, cutting use of ‘forever chemicals’
A new material developed by researchers from University of Toronto Engineering could offer a safer alternative to the nonstick chemicals commonly used in cookware and other applications. The new substance repels both water and [...]
