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Safety and efficacy studies of CRISPR-Cas9 treatment of sickle cell disease highlights disease-specific responses

22 Jul 2024

Abstract excerpt

Fetal hemoglobin (HbF) reactivation expression through CRISPR-Cas9 is a promising strategy for the treatment of sickle cell disease (SCD). Here, we describe a genome editing strategy leading to reactivation of HbF expression by targeting the binding sites (BSs) for the lymphoma-related factor (LRF) repressor in the γ-globin promoters. CRISPR-Cas9 treatment in healthy donor (HD) and patient-derived HSPCs resulted...

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PMID: 39044427
DOI: 10.1016/j.ymthe.2024.07.015

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