Gene Therapy for CNS and Sensory Organs

This lecture covers gene therapy for the central nervous system (CNS) and sensory organs, focusing on rare genetic diseases affecting these systems. It discusses the principles of gene therapy, the development of gene therapies for rare diseases, and the challenges and advancements in gene therapy strategies. The lecture also explores the use of viral vectors, nucleic acids, and precision medicine in treating inheritable and complex diseases. Specific examples include gene therapy for Spinal Muscular Atrophy, Amyotrophic Lateral Sclerosis, blindness, and deafness. The presentation delves into the history, current trends, and future prospects of gene therapy, emphasizing the potential applications in treating neurological disorders.