GeneticsbioRxivPreprint
Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies
Mégane Brusson, Anne Chalumeau, Pierre Martinucci…·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.
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