Preclinical Gene Therapy for Tuberous Sclerosis Complex - Research and Early Studies

Explore groundbreaking research on gene therapy for Tuberous Sclerosis Complex (TSC) in this 17-minute video featuring Dr. Xandra Breakefield and Dr. Edwina Abou Haidar from Mass General Hospital. Delve into the evolution of gene therapy, from identifying disease genes to gene replacement therapy, and its application to TSC patients. Learn about the role of Adeno-Associated Virus in gene therapy and understand TSC as an example of a tumor suppressor syndrome. Discover how gene therapy compares to everolimus in terms of survival rates, and its potential to reduce brain abnormalities and cell proliferation. Gain insights into the rationale behind pursuing gene therapy for TSC and the promising early studies conducted by these renowned researchers with over 40 years of experience in the field.