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For example, CRISPR-Cas9 editing to disrupt the BCL11A Erythroid Enhancer in HSPC was shown to reactivate production of fetal hemoglobin (HbF: 2 2 ) in place of adult hemoglobin (HbA: 2 2 ), and to overcome the transfusion-dependence in 91% (32/35) of -thalassemia patients in an international phase 3 trial comprising 20 (57%) TDT patients with severe genotypes 4 and in 2/2 pediatric 0 / 0 TDT patients in another recent clinical trial 5

doi:10.1067/mhn.2000.105415 Gamiz MJ, Lopez-Escamez JA

[DOI] [PubMed] [Google Scholar] 8.Benvenga S., Vicchio T., Di Bari F., Vita R., Fallahi P., Ferrari S.M., Catania S., Costa C., Antonelli A

Acta 243 , 8289 (2017)

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S2CID 44524962