Article

Multiplex base editing of <i>BCL11A</i> regulatory elements to treat sickle cell disease

2024-12-14

Abstract excerpt

Sickle cell disease (SCD) is a genetic anemia caused by the production of an abnormal adult hemoglobin. The clinical severity is lessened by elevated fetal hemoglobin (HbF) production in adulthood. A promising therapy is the transplantation of autologous, hematopoietic stem/progenitor cells (HSPCs) treated with CRISPR/Cas9 to downregulate the HbF repressor BCL11A via generation of double strand breaks (DSBs) in th...

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Literature Corpus work: 083b9edf-dcd2-5cc5-894a-ede216fa3f74
DOI: 10.1101/2024.12.13.628398

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