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Gene Therapy Insertional Mutagenesis Insights

15 Jan 2004

Abstract excerpt

In a milestone study describing the first “cure” of a genetic disease by retroviral gene therapy, 9 out of 10 infants born with X-linked severe combined immunodeficiency (SCID-X1, also known as γc or IL2RG deficiency) were successfully treated with autologous bone marrow stem cells infected ex

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Identifiers

PMID: 14726584
DOI: 10.1126/science.1091667

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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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