Co-Treatment of Erythroid Cells from β-Thalassemia Patients with CRISPR-Cas9-Based β⁰39-Globin Gene Editing and Induction of Fetal Hemoglobin.

ABSTRACT: Gene editing (GE) is an efficient strategy for correcting genetic mutations in monogenic hereditary diseases, including β-thalassemia. We have elsewhere reported that CRISPR-Cas9-based gene editing can be employed for the efficient correction of the β⁰39-thalassemia mutation. On the other hand, robust evidence demonstrates that the increased production of fetal hemoglobin (HbF) can be beneficial for patients with β-thalassemia. The aim of our study was to verify whether the de novo production of adult hemoglobin (HbA) using CRISPR-Cas9 gene editing can be combined with HbF induction protocols. The gene editing of the β⁰39-globin mutation was obtained using a CRISPR-Cas9-based experimental strategy; the correction of the gene sequence and the transcription of the corre

SUBMITTER: Cosenza LC 

PROVIDER: S-EPMC9601852 | biostudies-literature | 2022 Sep

REPOSITORIES: biostudies-literature