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Risk and Benefit Assessment of Gene Therapy with Lentiviral Vectors and Hematopoietic Stem Cells: The Skysona Case

1 Sept 2025

Abstract excerpt

Seven cases of hematological malignancy reported in recipients of Skysona™ (elivaldogene autotemcel) have reignited long-standing concerns about insertional mutagenesis in lentiviral vector (LV)-based gene therapy. Here, we dissect the molecular and clinical evidence underlying these events, place them in the broader context of over 300 patients treated with LV-modified hematopoietic stem and progenitor cells...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 40887817
DOI: 10.1177/10430342251372474

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026.

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