Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1-Reference-Cited by-同舟云学术

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Advancing gene targeting for primary immune deficiencies: Adenine base editing of the human IL2RG locus for correction of SCID-X1

Published:2024-06 Issue:6 Volume:32 Page:1606-1608
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

McIvor R. Scott^ORCID,Eaton Ella J.,Webber Beau R.,Moriarity Branden S.

Publisher

Elsevier BV

Reference15 articles.

1. Gene Therapies for Primary Immune Deficiencies;Kohn;Front. Immunol.,2021

2. Efficacy of gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N. Engl. J. Med.,2010

3. Gene therapy for immunodeficiency due to adenosine deaminase deficiency;Aiuti;N. Engl. J. Med.,2009

4. Restoring T and B cell generation in X-linked severe combined immunodeficiency mice through hematopoietic stem cells adenine base editing;Zhang;Mol. Ther.,2024

5. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency;Hacein-Bey-Abina;N. Engl. J. Med.,2003

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