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Article

Hematologic Cancer after Gene Therapy for Cerebral Adrenoleukodystrophy

9 Oct 2024

Abstract excerpt

Background Gene therapy with elivaldogene autotemcel (eli-cel) consisting of autologous CD34+ cells transduced with lentiviral vector containing ABCD1 complementary DNA (Lenti-D) has shown efficacy in clinical studies for the treatment of cerebral adrenoleukodystrophy. However, the risk of oncogenesis with eli-cel is unclear. Methods We performed integration-site analysis, genetic studies, flow cytometry, and...

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PMID: 39383458
DOI: 10.1056/nejmoa2405541

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