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Anemia is multifactorial, arising from defective CCO activity in erythrocyte precursors (normocytic anemia) and, in the case of TTM, from CP dysfunction, leading to iron retention and sideroblastic features [224, 244]

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For CRISPRa to work, single-guide RNAs (sgRNA) are designed to target regions between 1 to 1000 bp upstream of the transcription start site (TSS) within the promoter region of the target gene

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