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Gene Therapy for β-Hemoglobinopathies: From Discovery to Clinical Trials

9 Mar 2023

Abstract excerpt

Investigations to understand the function and control of the globin genes have led to some of the most exciting molecular discoveries and biomedical breakthroughs of the 20th and 21st centuries. Extensive characterization of the globin gene locus, accompanied by pioneering work on the utilization of viruses as human gene delivery tools in human hematopoietic stem and progenitor cells (HPSCs), has led to...

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PMID: 36992422
DOI: 10.3390/v15030713

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