CRISPR-Cas9-Based Gene Therapy for Treatment of IL2RA Deficiency

Ha, A; Shy, BR; Acevedo, L; Liu, W; Paruthiyil, S; Marrero-Rivera, G; Roth, TL; Catanzaro, J; Bluestone, JA; Tang, Q; Herold, K; Marson, A; Esensten, JH

MOLECULAR THERAPY, 2023; 31 (4): 717

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