Gene therapy shows promise for Alzheimer’s disease
VA Research News Briefs

Photo: ©iStock/YinYang
(06/04/2025)
San Diego VA researchers demonstrated in mouse models that targeting specific brain neurons through gene therapy in early-stage Alzheimer’s disease may help preserve memory and stimulate neuron growth. The researchers focused on the protein Cav-1, which promotes the growth and survival of connecting paths within the brain. They administered a synthetic copy of Cav-1, SynCav-1, to the brains of mice with an Alzheimer’s-like condition. SynCav-1 demonstrated the ability to preserve cognitive function and memory in the mice. Molecular analysis showed SynCav-1 protects neurons from degeneration and may even help grow new neuronal paths. The results suggest this type of gene therapy could be a promising new treatment for Alzheimer’s disease. (Signal Transduction and Targeted Therapy, May 28, 2025)