Matches in SemOpenAlex for { <https://semopenalex.org/work/W4387409872> ?p ?o ?g. }
- W4387409872 abstract "Lentiviral vector (LV)-based gene therapy holds promise for a broad range of diseases. Analyzing more than 280,000 vector integration sites (VISs) in 273 samples from 10 patients with X-linked severe combined immunodeficiency (SCID-X1), we discovered shared LV integrome signatures in 9 of 10 patients in relation to the genomics, epigenomics, and 3D structure of the human genome. VISs were enriched in the nuclear subcompartment A1 and integrated into super-enhancers close to nuclear pore complexes. These signatures were validated in T cells transduced with an LV encoding a CD19-specific chimeric antigen receptor. Intriguingly, the one patient whose VISs deviated from the identified integrome signatures had a distinct clinical course. Comparison of LV and gamma retrovirus integromes regarding their 3D genome signatures identified differences that might explain the lower risk of insertional mutagenesis in LV-based gene therapy. Our findings suggest that LV integrome signatures, shaped by common features such as genome organization, may affect the efficacy of LV-based cellular therapies." @default.
- W4387409872 created "2023-10-07" @default.
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- W4387409872 date "2023-10-06" @default.
- W4387409872 modified "2023-10-11" @default.
- W4387409872 title "Integrome signatures of lentiviral gene therapy for SCID-X1 patients" @default.
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