Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

doi:10.1172/JCI35798

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Article

Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients.

The Journal of clinical investigation - 1 Sept 2008

Howe Steven J, Mansour Marc R, Schwarzwaelder Kerstin, Bartholomae Cynthia, Hubank Michael, Kempski Helena, Brugman Martijn H, Pike-Overzet Karin, Chatters Stephen J, de Ridder Dick, Gilmour Kimberly C, Adams Stuart, Thornhill Susannah I, Parsley Kathryn L, Staal Frank J T, Gale Rosemary E, Linch David C, Bayford Jinhua, Brown Lucie, Quaye Michelle, Kinnon Christine, Ancliff Philip, Webb David K, Schmidt Manfred, von Kalle Christof, Gaspar H Bobby, Thrasher Adrian J

Abstract excerpt

X-linked SCID (SCID-X1) is amenable to correction by gene therapy using conventional gammaretroviral vectors. Here, we describe the occurrence of clonal T cell acute lymphoblastic leukemia (T-ALL) promoted by insertional mutagenesis in a completed gene therapy trial of 10 SCID-X1 patients. Integr...

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Identifiers

PMID: 18688286
DOI: 10.1172/JCI35798
PMCID: PMC2496964

Genes

LMO2
NOTCH1
STIL
TAL1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-18688286-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

Source receipt: PubMed via Helena Literature Corpus, retrieved 2 Sept 2026.

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