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

• Main Authors: Mégane Brusson (Author), Anne Chalumeau (Author), Pierre Martinucci (Author), Oriana Romano (Author), Tristan Felix (Author), Valentina Poletti (Author), Samantha Scaramuzza (Author), Sophie Ramadier (Author), Cecile Masson (Author), Giuliana Ferrari (Author), Fulvio Mavilio (Author), Marina Cavazzana (Author), Mario Amendola (Author), Annarita Miccio (Author)
• Format: Book
• Published: Elsevier, 2023-06-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...