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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| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Book |
| Published: |
Elsevier,
2023-06-01T00:00:00Z.
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