Developing disease-specific BBB-penetrating AAV vectors

Promising Gene Therapy Pathways for Combating Central Nervous System Diseases. Dr. Zhigang He, Professor of Neurology and Ophthalmology, Harvard Medical School, discusses how developing efficient BBB-penetrating recombinant AAV vectors shows great promise for enabling gene therapy for treating Central Nervous System (CNS) diseases including those relating to intellectual and developmental disabilities.