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Article

New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Nov 2007

Pike-Overzet Karin, van der Burg Mirjam, Wagemaker Gerard, van Dongen Jacques J M, Staal Frank J T

Abstract excerpt

The oncogenic potential of retrovirus-mediated gene therapy has been re-emphasized because four patients developed T-cell acute lymphoblastic leukemia (T-ALL)-like disease from an otherwise successful gene therapy trial for X-linked severe combined immunodeficiency (X-linked SCID). X-linked SCID, a disease caused by inactivating mutations in the IL2Rgamma gene, is part of a heterogeneous group of SCIDs...

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Source

Identifiers

PMID: 17726455
DOI: 10.1038/sj.mt.6300297

Genes

LMO2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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New insights and unresolved issues regarding insertional mutagenesis in X-linked SCID gene therapy.PMID 17726455Read ↗

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