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Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: A  Primary Immune Deficiency Treatment Consortium study.
Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SS, Deal CL, Kapoor N, Murguía-Favela L, Falcone EL, Prasad VK, Touzot F, Bleesing JJ, Chandrakasan S, Heimall JR, Bednarski JJ, Broglie LA, Chong HJ, Kapadia M, Prockop S, Dávila Saldaña BJ, Schaefer E, Bauchat AL, Teira P, Chandra S, Parta M, Cowan MJ, Dvorak CC, Haddad E, Kohn DB, Notarangelo LD, Pai SY, Puck JM, Pulsipher MA, Torgerson TR, Malech HL, Kang EM, Leiding JW. Grunebaum E, et al. J Allergy Clin Immunol. 2024 May;153(5):1423-1431.e2. doi: 10.1016/j.jaci.2024.01.013. Epub 2024 Jan 28. J Allergy Clin Immunol. 2024. PMID: 38290608
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease.
Schejtman A, Vetharoy W, Choi U, Rivat C, Theobald N, Piras G, Leon-Rico D, Buckland K, Armenteros-Monterroso E, Benedetti S, Ashworth MT, Rothe M, Schambach A, Gaspar HB, Kang EM, Malech HL, Thrasher AJ, Santilli G. Schejtman A, et al. Hum Gene Ther. 2021 Sep;32(17-18):949-958. doi: 10.1089/hum.2020.276. Epub 2021 May 6. Hum Gene Ther. 2021. PMID: 33740872 Free PMC article.