Biblio
Stem cell transplantation for primary immunodeficiency diseases: the North American experience. Curr Opin Allergy Clin Immunol. 2014.
. Progress in the field of hematopoietic stem cell-based therapies for inborn errors of immunity. Curr Opin Pediatr. 2023.
. Post-Transcriptional Genetic Silencing of to Treat Sickle Cell Disease. N Engl J Med. 2020.
Hematopoietic stem cell transplantation outcomes for 11 patients with dedicator of cytokinesis 8 deficiency. J Allergy Clin Immunol. 2016.
Hematopoietic stem cell transplantation is a curative therapy for transferrin receptor 1 (TFRC) deficiency. J Allergy Clin Immunol Pract. 2020.
. Fatal autoimmunity in mice reconstituted with human hematopoietic stem cells encoding defective FOXP3. Blood. 2015.
The diagnosis of severe combined immunodeficiency (SCID): The Primary Immune Deficiency Treatment Consortium (PIDTC) 2022 Definitions. J Allergy Clin Immunol. 2022.
The diagnosis of severe combined immunodeficiency: Implementation of the PIDTC 2022 Definitions. J Allergy Clin Immunol. 2022.
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.
Allogeneic hematopoietic cell transplantation is effective for p47phox chronic granulomatous disease: a PIDTC study. J Allergy Clin Immunol. 2024.