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Article

Persistent hematopoietic polyclonality after lentivirus-mediated gene therapy for Fabry disease

18 Jan 2023

Abstract excerpt

The safety and efficacy of lentivirus-mediated gene therapy was recently demonstrated in five male patients with Fabry disease—a rare X-linked lysosomal storage disorder caused by GLA gene mutations that result in multiple end-organ complications. To evaluate the risks of clonal dominance and leukemogenesis, which have been reported in multiple gene therapy trials, we conducted a comprehensive DNA insertion site...

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PMID: 36816757
DOI: 10.1016/j.omtm.2023.01.003

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