Keynote: AAV-mediated gene therapy to the central nervous system: prospect for Angelman syndrome

Quick Overview

Dr. Jim Wilson gave a keynote lecture at the 2017 FAST Science Summit on the prospects of AAV-mediated gene therapy for Angelman syndrome. He discussed the challenges of delivering genes to the central nervous system and the potential for gene therapy to treat the disease. Dr. Wilson highlighted the success of gene therapy in other diseases, such as spinal muscular atrophy, and the need for further research to determine the target cells and genes for Angelman syndrome. He also mentioned the importance of clinical trials and the potential for gene therapy to provide a permanent solution for patients. However, he emphasized the need for more research and collaboration to fully understand and develop effective gene therapy for Angelman syndrome.

Introduction

Dr. Jim Wilson, a pioneer in gene therapy, presented the keynote lecture at the 2017 FAST Science Summit. The author shares a personal story of how they connected with Dr. Wilson and highlights his expertise in developing viral vectors for gene therapy.

Background on Gene Therapy

Dr. Wilson provides an overview of gene therapy and its potential to treat rare diseases. He explains the challenges of delivering genes to the central nervous system and the importance of using viral vectors, specifically adeno-associated virus (AAV), to deliver the therapeutic genes.

Success in Other Diseases

Dr. Wilson discusses the success of gene therapy in treating inherited forms of blindness and hemophilia. He shares examples of animal models and clinical trials that have shown promising results in these diseases.

Prospects for Angelman Syndrome

Dr. Wilson acknowledges that there is still much to learn about Angelman syndrome and its underlying genetic defect. He emphasizes the need to define the target cells and determine the best gene to use for gene therapy. He also discusses the challenges of delivering the gene to the central nervous system and the importance of measuring success in clinical trials.

Timeline and Cost

Dr. Wilson explains that the timeline for moving gene therapy into human clinical trials depends on the progress of research and the community’s decision to move forward. He also discusses the cost of gene therapy and the potential for insurance coverage.

Conclusion

Dr. Wilson concludes by expressing his commitment to working with the Angelman syndrome community and leveraging the latest advancements in gene therapy to develop a treatment for the disease. He emphasizes the need for continued research and collaboration to ensure the best possible outcome for patients.