Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
Mégane Brusson, Anne Chalumeau, Pierre Martinucci, Oriana Romano, Valentina Poletti, Samantha Scaramuzza, Sophie Ramadier, Cécile Masson, Giuliana Ferrari, Fulvio Mavilio, Marina Cavazzana, Mario Amendola, Annarita Miccio
This study develops a bifunctional lentiviral vector for sickle cell disease that combines gene addition and gene silencing to reduce HbS levels and improve treatment efficacy.
bioRxiv · Dec 31, 2022 · PreprintGenetics