Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies

by Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano, Tristan Felix, Valentina Poletti, Samantha Scaramuzza, Sophie Ramadier, Cecile Masson, Giuliana Ferrari, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio
Published 2023-06-01

Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts

by Tui Neri, Sharon Muggeo, Marianna Paulis, Maria Elena Caldana, Laura Crisafulli, Dario Strina, Maria Luisa Focarelli, Francesca Faggioli, Camilla Recordati, Samantha Scaramuzza, Eugenio Scanziani, Stefano Mantero, Chiara Buracchi, Cristina Sobacchi, Angelo Lombardo, Luigi Naldini, Paolo Vezzoni, Anna Villa, Francesca Ficara
Published 2015-10-01