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Comment
. 2024 Apr 9;8(7):1817-1819.
doi: 10.1182/bloodadvances.2023012079.

Mending RAG2: gene editing for treatment of RAG2 deficiency

Affiliations
Comment

Mending RAG2: gene editing for treatment of RAG2 deficiency

Elizabeth D Hicks et al. Blood Adv. .
No abstract available

PubMed Disclaimer

Conflict of interest statement

Conflict-of-interest disclosure: M.D.K. is an author for Elsevier (UpToDate) and has received research funding from Chiesi Pharmaceuticals. E.D.H. declares no competing financial interests.

Comment on

References

    1. Pavel-Dinu M, Gardner CL, Nakauchi Y, et al. Genetically corrected RAG2-SCID human hematopoietic stem cells restore V(D)J-recombinase and rescue lymphoid deficiency. Blood Adv. 2024;8(7):1820–1833. - PubMed
    1. Dvorak CC, Haddad E, Buckley RH, et al. The genetic landscape of severe combined immunodeficiency in the United States and Canada in the current era (2010-2018) J Allergy Clin Immunol. 2019;143(1):405–407. - PMC - PubMed
    1. Lee YN, Frugoni F, Dobbs K, et al. A systematic analysis of recombination activity and genotype-phenotype correlation in human recombination-activating gene 1 deficiency. J Allergy Clin Immunol. 2014;133(4):1099–1108. - PMC - PubMed
    1. Delmonte OM, Villa A, Notarangelo LD. Immune dysregulation in patients with RAG deficiency and other forms of combined immune deficiency. Blood. 2020;135(9):610–619. - PMC - PubMed
    1. Schuetz C, Neven B, Dvorak CC, et al. SCID patients with ARTEMIS vs RAG deficiencies following HCT: increased risk of late toxicity in ARTEMIS-deficient SCID. Blood. 2014;123(2):281–289. - PMC - PubMed