New Gene Therapy for Alzheimer's May Preserve Cognitive Function: Study Shows

A study published in Signal Transduction and Targeted Therapy by researchers at the University of California San Diego School of Medicine introduces a gene therapy that may offer a powerful new approach to treating Alzheimer’s disease. Unlike current therapies that primarily target protein deposits in the brain, this innovative strategy focuses on altering the behavior of brain cells themselves to prevent damage and preserve cognitive function.

Current treatments for Alzheimer’s manage symptoms but do not stop the underlying disease process. The new gene therapy, developed by UC San Diego researchers, aims to halt or even reverse the progression of the disease by addressing its root causes.

In experiments using mice, the team delivered the gene therapy at the symptomatic stage of Alzheimer’s and found that it preserved hippocampal-dependent memory—a vital aspect of cognition that is typically impaired in patients. Notably, the treated mice exhibited gene expression patterns that closely resembled those of healthy mice, suggesting that the therapy may restore diseased cells to a more normal, functional state.

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.

Although additional studies are needed before the therapy can be tested in human trials, the findings represent a promising step toward a more effective and long-term solution for Alzheimer’s disease.

Reference: https://www.universityofcalifornia.edu/news/alzheimers-gene-therapy-shows-promise-preserving-cognitive-function#:~:text=This scan of a mouse,behavior of diseased brain cells.