The Promise of Gene-Based Therapies for Neurodegenerative Conditions

The ability to target the underlying cause of a disease and make a lasting correction makes gene therapy an attractive approach to treating neurodegenerative conditions. The advent of Zolgensma, a gene therapy for the treatment of the rare neurodegenerative condition spinal muscular atrophy, serves as a model for this approach. A recent review article in Nature Neuroscience looks at the advances in development of gene therapies for neurodegenerative disease and considers the challenges and promises. We spoke to article co-author Subhojit Roy, professor in the departments of Pathology and Neuroscience at the University of California, San Diego, about the pace of activity in this area, why he believes it is so promising, and its potential extend beyond monogenic diseases. This podcast is part of our ongoing Platforms of Hope series that explore advances in gene therapy and gene editing.