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

Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α2βS2). Transplantation of autologous hematopoietic stem and progenitor cells (HSPCs) transduced with lentiviral vectors (LVs) expressing an anti-sickling β-globin (βAS) is a p...

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Autori principali:	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
Natura:	Artigo
Lingua:	Inglês
Pubblicazione:	Elsevier 2023-06-01
Serie:	Molecular Therapy: Nucleic Acids
Soggetti:	MT: Non-coding RNAs sickle cell disease lentiviral vectors gene silencing gene therapy microRNA
Accesso online:	http://www.sciencedirect.com/science/article/pii/S2162253123000719
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