Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency.

Journal

Nature medicine

ISSN: 1546-170X

Titre abrégé: Nat Med

Pays: United States

ID NLM: 9502015

Informations de publication

Date de publication:
14 Feb 2024

Historique:

received: 31 08 2022

accepted: 21 12 2023

medline: 15 2 2024

pubmed: 15 2 2024

entrez: 15 2 2024

Statut: aheadofprint

Résumé

Adenosine deaminase (ADA) deficiency leads to severe combined immunodeficiency (SCID). Previous clinical trials showed that autologous CD34

Identifiants

DOI: 10.1038/s41591-023-02789-4 PMID: 38355973

pubmed: 38355973

doi: 10.1038/s41591-023-02789-4

pii: 10.1038/s41591-023-02789-4

doi:

Banques de données

ClinicalTrials.gov

['NCT03478670', 'NCT00598481']

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

Informations de copyright

Références

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