Gene Therapy Restores Memory and Cell Health in Alzheimer's Mice

With the global population ageing, the search for an effective Alzheimer's disease (AD) cure remains urgent. Recent research has highlighted the potential of Neurotrophic factor-α1/carboxypeptidase E (NF-α1/CPE) gene therapy. In a study involving mouse models, scientists delivered this therapeutic factor into the hippocampus—the brain's memory centre—and observed remarkable results: the treatment prevented memory loss, halted neurodegeneration, and stopped the activation of inflammatory microglial cells.

Crucially, the team discovered that a non-enzymatic form of the protein was just as effective, indicating the mechanism does not rely on enzymatic activity. To understand how this works, the researchers performed a quantitative proteomic analysis, examining changes in over 2,000 proteins. They found the therapy acts as a master regulator. It down-regulated proteins like Snx4 and Trim28, which are typically responsible for increasing toxic amyloid and tau levels in the brain.

The benefits extended beyond suppressing harmful elements. The therapy restored synaptic organisation, reviving the vital connections between neurons that are lost in AD. Furthermore, it reactivated autophagy—the cellular 'cleanup' mechanism—by up-regulating specific markers that are usually suppressed in the disease. This holistic restoration suggests that NF-α1/CPE could be a powerful tool for repairing the complex metabolic networks disrupted by Alzheimer's.