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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

Bioactive Materials, 32, 502-513

The supernatants were collected, and the pellets were resuspended in 0.2 N NaOH for protein determination

backing to consolidate his rule, not subordinating it to U.S