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Immunodeficiency: Gene therapy for primary immune deficiency

1 Sept 2024

Abstract excerpt

Current gene therapy for inborn errors of immunity have involved the use of gene addition approaches with viral delivery. This main strategy has had demonstrated success mainly in severe combined immune deficiency, Wiskott-Aldrich syndrome, and chronic granulomatous disease. Despite the increasing success of gene therapy, there are limitations of gene therapy, and, therefore, hematopoietic stem cell...

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PMID: 39294901
DOI: 10.2500/aap.2024.45.240054

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 3 Sept 2026.

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