Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies - Brusson, Mégane; Chalumeau, Anne; Martinucci, Pierre; Romano, Oriana; Felix, Tristan; Poletti, Valentina; Scaramuzza, Samantha; Ramadier, Sophie; Masson, Cecile; Ferrari, Giuliana; Mavilio, Fulvio; Cavazzana, Marina; et al. (Molecular therapy. Nucleic acids, 2023)
Keywords: gene addition; gene silencing; gene therapy; Human health and pathology; lentiviral vectors; Life Sciences; microRNA; MT: Non-coding RNAs; Original; sickle cell disease; βs silencing; βs silencing; lentiviral vectors; gene silencing; gene therapy; microRNA; gene addition; MT: Non-coding RNAs; sickle cell disease