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Exagamglogene autotemcel (exa-cel, formerly CTX001) CRISPR-Cas9 gene therapy was reported to increase fetal hemoglobin production through suppression of BCL11A expression and transfusion-independence in a 19-year-old female adult with TDT (beta 0 /beta + genotype) in a case report (183)

The compound reaches skin, hair follicles, internal organs, joints, and the nervous system

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