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Article

Transcription activator-like effector nuclease-mediated transduction of exogenous gene into IL2RG locus.

Scientific reports - 23 May 2014

Matsubara Yohei, Chiba Tomoki, Kashimada Kenichi, Morio Tomohiro, Takada Shuji, Mizutani Shuki, Asahara Hiroshi

Abstract excerpt

X-linked severe combined immunodeficiency (SCID-X1) caused by mutations in interleukin 2 receptor gamma (IL2RG) gene threatens the survival of affected boys during the first year of life unless hematopoietic stem cell transplantation is provided. Although viral vector-mediated gene therapy has been successfully performed in patients with no HLA-matched donors, leukemia caused by vector-mediated insertional...

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Source

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Identifiers

PMID: 24853770
DOI: 10.1038/srep05043
PMCID: PMC4031469

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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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