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CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease

1 Oct 2021

Abstract excerpt

Genome editing is potentially a curative technique available to all individuals with β-hemoglobinopathies, including sickle cell disease (SCD). Fetal hemoglobin (HbF) inhibits sickle hemoglobin (HbS) polymerization, and it is well described that naturally occurring hereditary persistence of HbF (HPFH) alleviates disease symptoms; therefore, reawakening of developmentally silenced HbF in adult red blood cells...

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PMID: 34729375
DOI: 10.1016/j.omtm.2021.09.010

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