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Article

Gene therapy for severe combined immunodeficiency: are we there yet?

The Journal of clinical investigation - 1 Jun 2007

Cavazzana-Calvo Marina, Fischer Alain

Abstract excerpt

Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone ma...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 17549248
DOI: 10.1172/JCI30953
PMCID: PMC1878528

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 2 Sept 2026. Prepared record publication-17549248-2026-09-02.2. Manifest publication-graph-cohort-2026-09-02.2.

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

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