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Current landscape of vector safety and genotoxicity after hematopoietic stem or immune cell gene therapy

8 Apr 2025

Abstract excerpt

Malignant transformation of gene modified haematopoietic stem cells caused anxiety following adverse events in early clinical trials using gamma-retroviral vectors (γRV) to correct haematopoietic stem cells (HSC) in monogenic immune disorders. Adoption of HIV-derived lentiviral vectors (LV) with SIN (self-inactivating) configurations greatly reduced risks and subsequently hundreds of patients have been dosed with...

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PMID: 40200078
DOI: 10.1038/s41375-025-02585-8

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