Innovative Gene Therapy for Alzheimer’s Disease Shows Potential in Protecting Cognitive Function

Researchers at the University of California San Diego School of Medicine have pioneered a groundbreaking gene therapy aiming to combat Alzheimer’s disease by targeting its fundamental causes. Unlike traditional treatments that mainly manage symptoms or focus on removing harmful protein deposits in the brain, this new approach seeks to modify the behavior of brain cells directly, potentially halting or reversing disease progression.

Alzheimer’s disease, which currently affects millions globally, is characterized by the buildup of abnormal proteins in the brain, leading to the death of neurons and a decline in memory and cognitive abilities. Current therapies can alleviate symptoms temporarily but do not stop the disease from advancing.

The innovative gene therapy was tested on mice models suffering from symptoms similar to human Alzheimer’s. Findings revealed that administering the treatment during the symptomatic stage helped preserve hippocampal-dependent memory, a vital component of cognition often impaired in Alzheimer’s patients. Furthermore, treated mice exhibited gene expression patterns akin to healthy, unaffected mice, indicating that the therapy might reprogram diseased cells for improved function.

While these promising results in animal studies mark a significant step forward, further research is essential to adapt and test this therapy in humans. Clinical trials will be necessary to evaluate safety, dosage, and effectiveness in patients.

The study was published in Signal Transduction and Targeted Therapy and was led by Dr. Brian Head, a professor of anesthesiology at UC San Diego, alongside co-senior researcher Dr. Shanshan Wang. Their work highlights a shift towards potentially transformative treatments that could address the root causes of Alzheimer’s, offering hope for more effective and lasting interventions against this devastating disease.

Source: https://medicalxpress.com/news/2025-06-alzheimer-gene-therapy-cognitive-function.html