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Lecture
Are you an EPFL student looking for a semester project?
Work with us on data science and visualisation projects, and deploy your project as an app on top of Graph Search.
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.