Principal Investigator
Miguel Sena-Esteves
Associate Professor
Miguel Sena-Esteves, PhD, is an associate professor of neurology and a member of the Horae Gene Therapy Center. Miguel Sena-Esteves received his PhD in biomedical sciences from the University of Porto, Portugal, where he started his work in gene therapy.
His laboratory at UMass Chan focuses on the development of adeno-associated virus (AAV) based gene therapies for lysosomal storage diseases, Huntington’s disease, ALS, FSHD and brain tumors. In addition, his laboratory develops novel AAV capsids to improve targeting, distribution and potent transduction of target tissues in these diseases, with a particular focus on the central nervous system.
Dr. Sena-Esteves leads the Tay-Sachs Gene Therapy Consortium with the focus to develop an AAV gene therapy and conduct the first-in-human clinical trial. He is also leading the pre-clinical development efforts for a GM1-gangliosidosis AAV gene therapy in collaboration with Lysogene. In 2011 he received the Outstanding New Investigator Award from the American Society of Gene & Cell Therapy (ASGCT) for his many contributions to the field of gene and cell therapy. He has authored 88+ peer-reviewed publications and is inventor on several patent applications.