Persistent Hematopoietic Polyclonality after Lentivirus-Mediated Gene Therapy for Fabry Disease

To evaluate the risks of clonal dominance and leukemogenesis, which have been reported in multiple gene therapy trials, investigators conducted a comprehensive DNA insertion site analysis of peripheral blood samples from the five patients in their gene therapy trial.
[Molecular Therapy-Methods & Clinical Development]
AbstractGraphical Abstract
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