Alzheimer’s Gene Therapy Reprograms Brain Cell Behavior

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.

Reference: Wang D, Chernov AV, Lam R, et al. Neuron-targeted caveolin-1 overexpression attenuates cognitive loss and pathological transcriptome changes in symptomatic Alzheimer’s disease models. Sig Transduct Target Ther. 2025;10(1):172. doi: 10.1038/s41392-025-02258-z

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