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Miguel Esteves
Associate Professor, Horae Gene Therapy Center at University of Massachusetts Medical School , USA


Miguel Sena-Esteves, PhD, studied Biochemistry at the Universidade do Porto (Portugal) and earned his PhD from the same University. His graduate studies focused on gene therapy for GM1 gangliosidosis and viral vector design. He conducted his post-doctoral training at Children’s Hospital of Philadelphia where he continued his work on lysosomal storage diseases (LSDs) and in vivo gene delivery. In 2004, he became an Assistant Professor at MGH/HMS in Boston where he started using AAV vectors for CNS gene therapy in LSDs and brain tumors. In 2009, he became Associate Professor at UMass Medical School. His laboratory focuses on AAV engineering for CNS gene transfer and therapeutic applications in neurodegenerative diseases with emphasis on LSDs (e.g., Tay-Sachs and  Sandhoff diseases, GM1 gangliosidosis), HD, ALS and brain tumors. He is part of a team of researchers and physicians developing AAV gene therapies for TSD, SD and GM1, with clinical trials initiated in 2019.