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Gene therapy for sickle cell disease: moving from the bench to the bedside

7 Jul 2021

Abstract excerpt

Gene therapy as a potential cure for sickle cell disease (SCD) has long been pursued, given that this hemoglobin (Hb) disorder results from a single point mutation. Advances in genomic sequencing have increased the understanding of Hb regulation, and discoveries of molecular tools for genome modification of hematopoietic stem cells have made gene therapy for SCD possible. Gene-addition strategies using gene...

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PMID: 34232993
DOI: 10.1182/blood.2019003776

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