Gene Therapy - Evolution and Therapeutic Applications (PDF)

Gene therapyis an attractive treatment strategy for both inherited and acquired late-onset neurodegenerative diseases (1). Over the past three decades, significant advances have been made in gene delivery methods and achieving more targeted and sustained expression of the gene of interest. The promising results obtained from preclinical studies have led to an increasing number of human clinical trials to investigate the safety and efficacy of gene therapy approaches for a broad range of disorders, in particular those involving the eye and the central nervous system.