UMass Medical School Faculty


Associate Professor Miguel Sena Esteves
Assiciate Professor Esteves earned his Ph.D. from the University of Porto in Portugal and did an NIH postdoctoral training program at the Center for Systems Biology at Massachusetts General Hospital/Harvard. He is a member of the Gene Therapy Center at UMass Medical School and the program manager of the international/interdisciplinary Tay-Sachs Gene Therapy Consortium. His research focuses on investigating gene therapy approaches for the treatment of neurodegenerative lysosomal storage diseases such as Tay-Sachs and Sandhoff diseases. His lab team is devising new ways to deliver therapeutic levels of the missing enzymes to the entire brain by injection of adeno-associated virus (AAV) vectors into specific structures in the central nervous system. He is also working on developing new AAV vectors capable of crossing the blood brain barrier for effective gene delivery to the brain after intravascular infusion.