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While the earlier clinical trials also had good response rates in consideration of patients who achieved transfusion independence, the patient cohorts treated in those studies had predominantly less severe β-thalassemia genotypes with 44% ( n = 8 of 18 patients) and 25% ( n = 1 of 4 patients) 3 as compared to the current study in which all 18 patients treated had severe genotypes

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One of the weaknesses of this study is that the gene and protein expression reported are based on a single time point of 6 weeks