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TALEN-Mediated Gene Editing of HBG in Human Hematopoietic Stem Cells Leads to Therapeutic Fetal Hemoglobin Induction

31 Dec 2018

Abstract excerpt

Elements within the γ-hemoglobin promoters ( HBG1 and HBG2 ) function to bind transcription complexes that mediate repression of fetal hemoglobin expression. Sickle cell disease (SCD) subjects with a 13-bp deletion in the HBG1 promoter exhibit a clinically favorable hereditary persistence of fetal hemoglobin (HPFH) phenotype. We developed TALENs targeting the homologous HBG promoters to de-repress fetal...

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Share to Epigenetics and DNA Methylation
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PMID: 30705922
DOI: 10.1016/j.omtm.2018.12.008

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