Alzheimer’s gene therapy is consolidating as the most viable hope to halt the advance of this neurodegenerative disease, according to a new development by the Biomedical Research Center of La Rioja (CIBIR). A team of researchers in Logroño has initiated an innovative project that seeks to identify and modify an essential protein associated with the development of Alzheimer’s, attempting to prevent the disease from its initial stages.
Led by CIBIR, the project focuses on direct genetic modification to slow down neuronal death. Although Alzheimer’s gene therapy applied in humans still requires years of validation, preliminary studies in animal models have shown promising results. The strategy involves inoculating a virus carrying a specific gene into the brain, capable of reducing the formation of amyloid plaques, one of the most characteristic signs of the pathology.
Benefits observed in preclinical models
Recent studies in mice with early-stage Alzheimer’s disease revealed that administration of the gene managed to significantly halt its progression. After four months, the treated subjects presented few amyloid plaques compared to the untreated ones, whose brains showed multiple deposits. Furthermore, Alzheimer’s gene therapy preserved memory at levels comparable to healthy mice and prevented the loss of brain cells in the hippocampus.
Limitations and future of Alzheimer’s gene therapy
Despite the advances, direct application in people presents significant technical challenges. Alzheimer’s gene therapy requires direct injection of genes into the brain, a complex procedure currently limited to preclinical models. Experts such as those from Imperial College London and the Autonomous University of Barcelona have confirmed that, although its mass use in humans remains far off, the results open doors to new future treatments.
Research continues with the goal of developing safer delivery systems, such as brain-targeted extracellular vesicles, so that Alzheimer’s gene therapy can become a real option for patients. While other approaches like CRISPR gene editing and CAR-T cells also show potential, CIBIR’s innovation represents a crucial step in the prevention of neurodegenerative diseases.
In conclusion, Alzheimer’s gene therapy offers tangible hope to halt the development of this disease, although its implementation in humans will depend on future clinical trials confirming its safety and efficacy.
