Miguel Sena-Esteves

Miguel Sena-Esteves, PhD, is an Associate Professor in the Neurology Department and Horae Gene Therapy Center at UMass Chan Medical School. His laboratory focuses on AAV capsid engineering for in vivo gene transfer to CNS and other tissues, as well as gene therapies for various inherited diseases including lysosomal storage diseases, Huntington’s disease, amyotrophic lateral sclerosis, neurofibromatosis type 1 (NF1), and various other neurological and muscular dystrophies. He is part of a team of researchers and physicians that developed AAV gene therapies for GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), and GM1 gangliosidosis both currently being tested in phase I/II clinical trials. His goal is to continue to develop gene therapies for rare diseases. He is also the director of the newly created Translational Institute for Molecular Therapeutics at UMass Chan where the goal is to assist researchers in the rapid translation of their findings in the laboratory to first-in-human clinical trials.