The Bradbury Laboratory is dedicated to understanding disease mechanisms and developing therapeutic approaches for rare, pediatric neurodegenerative disorders. Advances in adeno-associated virus (AAV) vectors have led to safer and more efficient viral vehicles to deliver therapeutic transgenes in a single injection, and gene therapy is now a favorable therapeutic intervention for monogenic diseases.  A primary focus of the Bradbury lab is improving AAV targeting of myelinating cells, a current limitation of AAV, in order to advance therapeutic outcomes for leukodystrophies and other white matter disorders. The Bradbury lab is translational in nature with a commitment to safely and efficiently moving therapies into the clinic for rare, pediatric disorders.