AAMS Genetics & Molecular Medicine · Vol. 1 · Issue 1 · 2024-03-15
CRISPR-Cas12a Mediated Gene Editing for Sickle Cell Disease: A Preclinical Study
Park JH, Kim SY, Lee DW
Department of Medical Genetics, Seoul National University Hospital, Seoul, South Korea¹; Division of Hematology, Samsung Medical Center, Seoul, South Korea²
Abstract
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
Full Text
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.
This article is published open access by AAMS Genetics & Molecular Medicine. The complete peer-reviewed text can be read on the journal page.
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