Biblio

Author Title Type [ Year(Asc)]
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2024
Grunebaum E, Arnold DE, Logan B, Parikh S, Marsh RA, Griffith LM, Mallhi K, Chellapandian D, Lim SSi, Deal CL, et al. Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: a PIDTC study. J Allergy Clin Immunol. 2024.
Keller MD, Hanley PJ, Chi Y-Y, Aguayo-Hiraldo P, Dvorak CC, Verneris MR, Kohn DB, Pai S-Y, Saldaña BJDávila, Hanisch B, et al. Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. Nat Commun. 2024;15(1):3258.
2023
Labrosse R, Chu J, Armant M, Everett JK, Pellin D, Kareddy N, Frelinger AL, Henderson LA, O'Connell AE, Biswas A, et al. Outcomes of Hematopoietic Stem Cell Gene Therapy for Wiskott-Aldrich Syndrome. Blood. 2023.
Arnold DE, Pai S-Y. Progress in the field of hematopoietic stem cell-based therapies for inborn errors of immunity. Curr Opin Pediatr. 2023.
2022
Dvorak CC, Haddad E, Heimall J, Dunn E, Cowan MJ, Pai S-Y, Kapoor N, Satter LForbes, Buckley RH, O'Reilly RJ, et al. The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. J Allergy Clin Immunol. 2022.
Dvorak CC, Haddad E, Heimall J, Dunn E, Buckley RH, Kohn DB, Cowan MJ, Pai S-Y, Griffith LM, Cuvelier GDE, et al. The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. J Allergy Clin Immunol. 2022.
2020
Whangbo JS, Chou J, Al-Dhekri H, Harris M, Geha RS, Pai S-Y, Al-Herz W. Hematopoietic stem cell transplantation is a curative therapy for transferrin receptor 1 (TFRC) deficiency. J Allergy Clin Immunol Pract. 2020.
Esrick EB, Lehmann LE, Biffi A, Achebe M, Brendel C, Ciuculescu MF, Daley H, MacKinnon B, Morris E, Federico A, et al. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
2016
Al-Herz W, Chu JI, van der Spek J, Raghupathy R, Massaad MJ, Keles S, Biggs CM, Cockerton L, Chou J, Dbaibo G, et al. Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016.
2015
Goettel JA, Biswas S, Lexmond WS, Yeste A, Passerini L, Patel B, Yang S, Sun J, Ouahed J, Shouval DS, et al. Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3. Blood. 2015.
2014
Pai S-Y, Cowan MJ. Stem cell transplantation for primary immunodeficiency diseases: the North American experience. Curr Opin Allergy Clin Immunol. 2014.
2013
Leen AM, Bollard CM, Mendizabal AM, Shpall EJ, Szabolcs P, Antin JH, Kapoor N, Pai S-Y, Rowley SD, Kebriaei P, et al. Multicenter study of banked third party virus-specific T-cells to treat severe viral infections after hematopoietic stem cell transplantation. Blood. 2013.