Research alert: Alzheimer's gene therapy shows promise in preserving cognitive function

By reprogramming the behavior of brain cells, new gene therapy could stop Alzheimer’s at the source

2025-06-05

(Press-News.org) Researchers at University of California San Diego School of Medicine have developed a gene therapy for Alzheimer’s disease that could help protect the brain from damage and preserve cognitive function. Unlike existing treatments for Alzheimer’s that target unhealthy protein deposits in the brain, the new approach could help address the root cause of Alzheimer’s disease by influencing the behavior of brain cells themselves.

Alzheimer's disease affects millions of people around the world and occurs when abnormal proteins build up in the brain, leading to the death of brain cells and declines in cognitive function and memory. While current treatments can manage symptoms of Alzheimer’s, the new gene therapy aims to halt or even reverse disease progression.

Studying mice, the researchers found that delivering the treatment at the symptomatic stage of the disease preserved hippocampal-dependent memory, a critical aspect of cognitive function that is often impaired in Alzheimer's patients. Compared to healthy mice of the same age, the treated mice also had a similar pattern of gene expression, suggesting that the treatment has the potential to alter the behavior of diseased cells to restore them to a healthier state.

While further studies will be required to translate these findings into human clinical trials, the gene therapy offers a unique and promising approach to mitigating cognitive decline and promoting brain health.

The study, published in Signal Transduction and Targeted Therapy, was led by senior author Brian Head, M.D., professor of anesthesiology at UC San Diego School of Medicine and Veterans Affairs research career scientist, and co-senior author Shanshan Wang, M.D. Ph.D., an assistant professor of anesthesiology at UC San Diego School of Medicine. The gene therapy technology was licensed by UC San Diego to Eikonoklastes Therapeutics in 2021. Eikonoklastes was granted Orphan Drug Designation (ODD) by the FDA for the use of the patented gene therapy in amyotrophic lateral sclerosis (ALS) also known as Lou Gehrig’s disease.

# # #

END

ELSE PRESS RELEASES FROM THIS DATE:

New York Eye and Ear Infirmary of Mount Sinai opens first Comprehensive Center for Refractive Solutions in New York

2025-06-05

New York Eye and Ear Infirmary of Mount Sinai (NYEE) has announced the opening of the Center for Refractive Solutions—the first in New York City and one of few in the United States—that offers comprehensive and innovative treatment options for those who require refractive correction. This includes premium cataract and intraocular lens surgery, and corneal refractive procedures such as LASIK, to reduce patients’ dependency on glasses and contact lenses. The newly renovated space, located at 310 East 14th Street in Lower Manhattan, is solely dedicated ...

Storm ready: FAU Sensing Institute’s weather network delivers real-time forecasting

2025-06-05

As Floridians prepare for an active 2025 Atlantic hurricane season, the most serious threat may not come from wind, but from water. Data from the National Hurricane Center shows that 86% of all direct hurricane and tropical storm fatalities in the United States between 2013 and 2023 were caused by water impacts – freshwater flooding, storm surge and rip currents. Of those, more than half resulted from drownings due to inland flooding, highlighting the critical importance of accurate rainfall and flood forecasting. Florida Atlantic University’s Sensing Institute (I-SENSE) has emerged as a vital contributor to the nation’s ...

UChicago receives $21 million to establish visionary center in quantum engineering and health

2025-06-05

A $21 million gift from philanthropist Thea Berggren to the University of Chicago will establish the Berggren Center for Quantum Biology and Medicine, launching a bold scientific field that merges quantum technology with biology to transform the future of medicine. This pioneering, interdisciplinary effort seeks to harness the power of quantum engineering — capable of the most sensitive measurements known to science — to peer inside the human body in unprecedented ways. The goal is to unlock insights into biology and disease that were previously out of reach, ...

Inherited genetic trait predicts resistance to immunotherapy for deadly skin cancer

2025-06-05

Tests in 1,225 patients with the most deadly form of skin cancer reveal for the first time a genetic trait among most of those who did not respond to the latest cancer treatments, known as immune checkpoint inhibitors. Metastatic melanoma, as the disease is formally named, kills nearly 10,000 Americans annually. While the drugs have proven highly successful in treating metastatic melanoma and several other cancers, the therapies are known to not work for almost half of those who are prescribed them, usually after initial chemotherapy or surgery have failed ...

Oxford physicists recreate extreme quantum vacuum effects

2025-06-05

Physicists at the University of Oxford have successfully simulated how light interacts with empty space—a phenomenon once thought to belong purely to the realm of science fiction. The simulations recreated a bizarre phenomenon predicted by quantum physics, where light appears to be generated from darkness. The findings pave the way for real-world laser facilities to experimentally confirm bizarre quantum phenomena. The results have been published today (5 June) in Communications Physics. Using advanced computational modelling, a research team led by the University of Oxford, working in partnership with the Instituto Superior Técnico in the University of Lisbon, ...

Talking therapy could be effective treatment for stroke survivors

2025-06-05

Stroke survivors with depression or anxiety who attend talking therapy sessions are more likely to recover from their psychological symptoms, finds a new study by UCL researchers. The first of its kind study, published in Nature Mental Health, analysed data from all 1.9 million patients who attended NHS Talking Therapies for Anxiety and Depression services in England between 2012 and 2019, including 7,597 stroke survivors. More than one in three stroke survivors experience depression or anxiety. Without appropriate mental health treatment, ...

A new method for studying mechanical proteins and their involvement in muscular disorders

2025-06-05

A team at the Centro Nacional de Investigaciones Cardiovasculares (CNIC), led by Jorge Alegre-Cebollada, has developed an innovative method, called TEVs-TTN, for studying the specific mechanical functions of proteins through their controlled cleavage, a process that renders the proteins unable to sense and transmit mechanical force. The study results extend knowledge about the development of muscular diseases. The study, published in Nature Biomedical Engineering, demonstrates that interrupting mechanical transmission by the protein titin precipitates muscular diseases. This finding opens new routes to understanding muscular dystrophies and other diseases ...

Two big events shaped the herbivores during 60 million years but their role remains

2025-06-05

Two major environmental shifts have triggered global transformations in large herbivore communities. A new study with researchers from the University of Gothenburg show how these ecosystems remained remarkably resilient despite extinction and upheaval. From mastodons to ancient rhinos and giant deer, large herbivores have been shaping Earth’s landscapes for millions of years. A new study, published in Nature communications, shows how these giants responded to dramatic environmental shifts – and how their ecosystems found ways to stay together, even as species disappeared. An ...

TU Graz study: front brake lights could significantly reduce the number of road accidents

2025-06-05

The idea of the front brake light has been around for some time, but no vehicle manufacturer has as yet implemented it. A research team led by Ernst Tomasch from the Institute of Vehicle Safety at Graz University of Technology (TU Graz) in collaboration with the Bonn Institute for Legal and Traffic Psychology (BIRVp) has now analysed their effect on road safety in an accident reconstruction study. The analysis of 200 real accidents at road junctions showed that – depending on the reaction time of road ...

A new mathematical model helps European regions set suitable targets to close gender gaps in education

2025-06-05

The gender gap in education doesn’t always disadvantage women. In countries like Estonia, Iceland, or Sweden, women outperform men in key indicators such as tertiary education and lifelong learning. But that, too, is a gender gap. That’s the starting point for researchers at the Miguel Hernández University of Elche (UMH), who have developed a mathematical model to support European education authorities in improving performance and reducing gender disparities, regardless of which group is underperforming. “In many European ...

LAST 30 PRESS RELEASES:

Plan, prepare, conquer: predicting mountain accident risks with deep learning and pre-climb data

New ancient marine reptile species discovered in Germany's famous Jurassic fossil beds

Psychedelics and non-hallucinogenic analogs work through the same receptor, up to a point

The Lancet: Plastic pollution is an underrecognised threat to health, experts warn as they launch a project to track plastics’ health impacts and monitor progress

The Lancet Countdown on Health and Plastics to track impact of plastic production and pollution on human health

Announcing The Lancet Countdown on Health and Plastics

Study unexpectedly finds living in rural, rather than urban environments in first five years of life could be a risk factor for developing type 1 diabetes