Alzheimer’s disease (AD) remains a significant challenge due to its complex pathology and limited effectiveness of current treatments. Recent advances in gene therapy offer promising avenues for addressing AD by targeting its root causes. Studies highlight the role of genetic mutations, such as those in the PSEN-1, PSEN-2, and APP genes, in familial AD, as well as mutations in the tau and APO-E genes, which contribute to amyloid accumulation, tau hyperphosphorylation, and neurodegeneration. Gene-based interventions, focusing on correcting these genetic abnormalities, have shown potential in animal models and early clinical trials to mitigate cognitive decline and neuronal loss, paving the way for innovative treatments.

Gene therapy, combined with multimodal strategies like lifestyle interventions and advanced drug delivery mechanisms, represents a shift toward more comprehensive treatment approaches. By leveraging genetic insights and animal models, researchers aim to identify novel therapeutic targets and enhance the precision of gene delivery techniques. The integration of gene-based strategies with traditional and non-pharmacological methods underscores a transformative direction in AD treatment, offering hope for improved outcomes in this progressive neurodegenerative disease.

Reference: Ataei B, Hokmabadi M, Asadi S, et al. A review of the advances, insights, and prospects of gene therapy for Alzheimer’s disease: A novel target for therapeutic medicine. Gene. 2024 Jun 20;912:148368. doi: 10.1016/j.gene.2024.148368. Epub 2024 Mar 12. PMID: 38485038.