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Article

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.

The Journal of clinical investigation - 1 Sept 2008

Hacein-Bey-Abina Salima, Garrigue Alexandrine, Wang Gary P, Soulier Jean, Lim Annick, Morillon Estelle, Clappier Emmanuelle, Caccavelli Laure, Delabesse Eric, Beldjord Kheira, Asnafi Vahid, MacIntyre Elizabeth, Dal Cortivo Liliane, Radford Isabelle, Brousse Nicole, Sigaux François, Moshous Despina, Hauer Julia, Borkhardt Arndt, Belohradsky Bernd H, Wintergerst Uwe, Velez Maria C, Leiva Lily, Sorensen Ricardo, Wulffraat Nicolas, Blanche Stéphane, Bushman Frederic D, Fischer Alain, Cavazzana-Calvo Marina

Abstract excerpt

Previously, several individuals with X-linked SCID (SCID-X1) were treated by gene therapy to restore the missing IL-2 receptor gamma (IL2RG) gene to CD34+ BM precursor cells using gammaretroviral vectors. While 9 of 10 patients were successfully treated, 4 of the 9 developed T cell leukemia 31-68 months after gene therapy. In 2 of these cases, blast cells contained activating vector insertions near the LIM...

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Source

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Identifiers

PMID: 18688285
DOI: 10.1172/JCI35700
PMCID: PMC2496963

Genes

LMO2
BMI1
CCND2
NOTCH1
TAL1

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1.PMID 18688285Read ↗

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