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

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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Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: Elsevier 2023-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: Non-coding RNAs
sickle cell disease
lentiviral vectors
gene silencing
gene therapy
microRNA
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253123000719

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http://www.sciencedirect.com/science/article/pii/S2162253123000719

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