Article

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

2022-12-31

Abstract excerpt

Sickle cell disease (SCD) is due to a mutation in the β-globin ( HBB ) gene causing the production of the toxic sickle hemoglobin (HbS, a 2 β S 2 ). Transplantation of autologous hematopoietic stem/progenitor cells (HSPCs) transduced with lentiviral vectors (LVs) expressing an anti-sickling β-globin (βAS) is a promising treatment; however, it is only partially effective and patients still present elevated HbS l...

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Literature Corpus work: 33272160-ae97-5036-8260-9d65b7115e0f
DOI: 10.1101/2022.12.31.522279

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