The GATA1-HS2 enhancer allows persistent and position-independent expression of a β-globin transgene.

by Annarita Miccio, Valentina Poletti, Francesca Tiboni, Claudia Rossi, Antonella Antonelli, Fulvio Mavilio, Giuliana Ferrari
Published 2011-01-01

Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-Thalassemia

by Maria Rosa Lidonnici, Ylenia Paleari, Francesca Tiboni, Giacomo Mandelli, Claudia Rossi, Michela Vezzoli, Annamaria Aprile, Carsten Werner Lederer, Alessandro Ambrosi, Franck Chanut, Francesca Sanvito, Andrea Calabria, Valentina Poletti, Fulvio Mavilio, Eugenio Montini, Luigi Naldini, Patrizia Cristofori, Giuliana Ferrari
Published 2018-12-01