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Genome editing for primary immunodeficiencies: A therapeutic perspective on Wiskott-Aldrich syndrome.
Front Immunol. 2022 Aug 18;13:966084. doi: 10.3389/fimmu.2022.966084. eCollection 2022.
Front Immunol. 2022.
PMID: 36059471
Free PMC article.
Review.
CRISPR/Cas9-Based Disease Modeling and Functional Correction of Interleukin 7 Receptor Alpha Severe Combined Immunodeficiency in T-Lymphocytes and Hematopoietic Stem Cells.
Rai R, Steinberg Z, Romito M, Zinghirino F, Hu YT, White N, Naseem A, Thrasher AJ, Turchiano G, Cavazza A.
Rai R, et al.
Hum Gene Ther. 2024 Apr;35(7-8):269-283. doi: 10.1089/hum.2023.100. Epub 2024 Feb 29.
Hum Gene Ther. 2024.
PMID: 38251667
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