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Article

Correction of murine Rag2 severe combined immunodeficiency by lentiviral gene therapy using a codon-optimized RAG2 therapeutic transgene.

Molecular therapy : the journal of the American Society of Gene Therapy - 1 Oct 2012

van Til Niek P, de Boer Helen, Mashamba Nomusa, Wabik Agnieszka, Huston Marshall, Visser Trudi P, Fontana Elena, Poliani Pietro Luigi, Cassani Barbara, Zhang Fang, Thrasher Adrian J, Villa Anna, Wagemaker Gerard

Abstract excerpt

Recombination activating gene 2 (RAG2) deficiency results in severe combined immunodeficiency (SCID) with complete lack of T and B lymphocytes. Initial gammaretroviral gene therapy trials for other types of SCID proved effective, but also revealed the necessity of safe vector design. We report the development of lentiviral vectors with the spleen focus forming virus (SF) promoter driving codon-optimized human...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 22692499
DOI: 10.1038/mt.2012.110
PMCID: PMC3464632

Genes

RAG2

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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