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Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease

19 Aug 2021

Abstract excerpt

Sickle cell disease (SCD) is caused by a mutation in the β-globin gene leading to polymerization of the sickle hemoglobin (HbS) and deformation of red blood cells. Autologous transplantation of hematopoietic stem/progenitor cells (HSPCs) genetically modified using lentiviral vectors (LVs) to express an anti-sickling β-globin leads to some clinical benefit in SCD patients, but it requires high-level transgene...

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PMID: 34418541
DOI: 10.1016/j.ymthe.2021.08.019

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