Recently, we have seen strong progress in the application of gene therapy for rare diseases, including inherited forms of blindness, hemophilia and lysosomal disorders such as Pompe disease. Another emerging area for gene therapy are CNS disorders including monogenic disorders and beyond to more complex neurodegenerative diseases, including Parkinson’s and Alzheimer’s. The challenges with CNS gene therapy research include the pathophysiological complexity of CNS diseases and access to and delivery across the blood-brain barrier. This panel will explore some of the exciting work that is being done to identify the right platform and vehicle to access the brain, as well as to select the right targets to deliver clear clinical outcomes.
Session ID: 547916