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Article

Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy

24 Jul 2006

Abstract excerpt

Although gene therapy can cure patients with severe combined immunodeficiency (SCID) syndromes, the clinical occurrence of T cell malignancies due to insertional mutagenesis has raised concerns about the safety of gene therapy. Several key questions have remained unanswered: (i) are there unique risk factors for X-linked SCID (XSCID) gene therapy that increase the risk of insertional mutagenesis; (ii) what other...

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PMID: 16864781
DOI: 10.1073/pnas.0603635103

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