San Raffaele Telethon Institute for Gene Therapy
Elena Barbon, PhD, San Raffaele Telethon Institute for Gene Therapy, IRCCS San Raffaele Scientific Institute; Milan,Italy
Elena Barbon is currently a post-doctoral researcher at the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) in Milan, Italy. She earned her PhD from the University of Ferrara, Italy, in 2015, under the supervision of Prof.Mirko Pinotti. In her thesis work, she focused on the characterization of haemophilia-causing splicing mutations and their correction exploiting small modified RNAs delivered via AAV vectors in a mouse model. Moreover, she developed synthetic transcription factors as a therapeutic approach for coagulation disorders caused by mutations in the promoter. After completing the PhD, she moved to Genethon Institute, France, to work in Dr.Federico Mingozzi’s immunology and liver diseases laboratory. During that time, she developed innovative gene therapy approaches for coagulation disorders, i.e haemophilia and von Willebrand disease, exploiting AAV-based corrective strategies. She also participated in projects focused on the study of the influence of pre-existing antibodies on AAV vector transduction and on the development of strategies aimed at overcoming the pre-existing immunity against the AAV vector, which still represents one of the main challenges in AAV gene therapy field. Her current work in SR-TIGET is focused on the development of liver directed gene therapy approaches for inherited metabolic diseases.
Tuesday, July 12, 2022
8:00 AM – 8:20 AM