This preclinical study evaluates the efficacy and safety of CRISPR-Cas12a-based gene editing targeting the BCL11A enhancer in CD34+ hematopoietic stem cells for sickle cell disease treatment. Results demonstrate 89% editing efficiency with sustained fetal hemoglobin induction.
Keywords: CRISPR-Cas12a, Sickle Cell Disease, Gene Editing, Hematopoietic Stem Cells, Fetal Hemoglobin