Article

LNP-mediated <i>BCL11A</i> Editing Corrects Sickling Phenotypes and Preserves HSPC Fitness Compared to Electroporation

2026-08-27

Abstract excerpt

<h4>ABSTRACT</h4> Hemoglobinopathies, including sickle cell disease (SCD) and thalassemia syndromes, affect millions of individuals worldwide who have limited access to curative therapies. Autologous hematopoietic stem cell transplant following ex vivo CRISPR editing of the BCL11A erythroid enhancer reactivates fetal hemoglobin (HbF) and achieves an effective cure, but the resource constraints of clinically app...

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Literature Corpus work: b78b7fc8-dc4d-55b7-b319-44628522602a
DOI: 10.64898/2026.08.26.747413

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