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Cardiovascular safety A thorough QT study found no clinically relevant QTc prolongation, even at supratherapeutic doses

Int J Mol Sci 2023;24:14858

10.1172/JCI106569 [DOI] [PMC free article] [PubMed] [Google Scholar] 19

Clinical trials using lentiviral vectors to introduce functional beta-globin genes into a patients own hematopoietic stem cells have shown promising results, with some individuals achieving transfusion independence