The promise of gene therapy in common types of dementia.

Abstract

Dementia is an umbrella term describing different types of diseases that lead to cognitive impairment and memory dysfunction, predominantly affecting older adults. The most common forms include Alzheimer's disease (AD), vascular dementia (VaD), dementia with Lewy bodies (DLB), and frontotemporal dementia (FTD). Despite extensive research, there is no definitive cure for dementia, primarily due to its complex and multifactorial nature, particularly the role of genetic abnormalities. Gene therapy, a novel therapeutic approach, aims to correct defective genes or introduce functional gene products by delivering specific DNA sequences to patients, and is often considered for individuals unresponsive to conventional treatments, such as those with dementia. Over the past decade, significant research has explored the potential of gene therapy in dementia, offering new hope for more effective treatments. However, several challenges remain in its practical application. One key challenge is developing safe and efficient gene delivery methods, as the brain's intricate structure and protective barriers present significant obstacles. Furthermore, ensuring the long-term expression and stability of therapeutic genes is crucial for sustained benefit. Future studies should focus on identifying genes implicated in different types of dementia, optimizing gene delivery systems, improving gene-targeting specificity, and conducting comprehensive clinical trials to assess the safety and efficacy of these therapies. Addressing these challenges could pave the way for novel treatment strategies, ultimately improving the quality of life for individuals with dementia.