加州大学圣地亚哥分校的科学家开发出一种突破性的基因疗法，不仅能掩盖阿尔茨海默病症状，还可能真正恢复大脑功能。在动物实验中，该疗法有效保护了小鼠的记忆能力，并通过调控机制使病变脑细胞恢复了接近健康细胞的功能特性。
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, Ph.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.