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Nature Genetics|August 18, 2014
JAGN1 deficiency causes aberrant myeloid cell homeostasis and congenital neutropeniaKaan Boztug, Päivi M Järvinen, Elisabeth Salzer, et al.
Bone Marrow Transplantation|March 25, 2022
Allogeneic hematopoietic stem cell transplantation for adult HLH: a retrospective study by the chronic malignancies and inborn errors working parties of EBMTRafal Machowicz, Felipe Suarez, Wieslaw Wiktor-Jedrzejczak, et al.
Blood Advances|June 20, 2025
Allogeneic hematopoietic stem cell transplantation for STAT3 hyper-IgE syndrome: a worldwide studyChristo Tsilifis, Johannes Raedler, Joanna Renke, et al.
Blood Advances|March 3, 2026
Outcome of Second Conditioned Allogeneic Stem Cell Transplant in Children with Non-SCID Inborn Errors of ImmunityZohreh Nademi, Su-Han Lum, Patrick Gilbert, et al.
Blood|January 31, 2022
Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysisMichael H Albert, Mary A Slatter, Andrew R Gennery, et al.
The Lancet. Haematology|April 15, 2019
Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical studyFrancesca Ferrua, Maria Pia Cicalese, Stefania Galimberti, et al.
Clinical Cancer Research : an Official Journal of the American Association for Cancer Research|October 21, 2020
Hematopoietic Stem Cell Transplantation Positively Affects the Natural History of Cancer in Nijmegen Breakage SyndromeBeata Wolska-Kusnierz, Agata Pastorczak, Wojciech Fendler, et al.
The Journal of Allergy and Clinical Immunology|September 24, 2016
A prospective study on the natural history of patients with profound combined immunodeficiency: An interim analysisCarsten Speckmann, Sam Doerken, Alessandro Aiuti, et al.
Blood|July 3, 2020
Hematopoietic cell transplantation in chronic granulomatous disease: a study of 712 children and adultsRobert Chiesa, Junfeng Wang, Henric-Jan Blok, et al.
Blood|September 22, 2025
Hematopoietic stem cell transplantation for purine nucleoside phosphorylase deficiency: an EBMT-IEWP retrospective studyUli S Herrmann, Matthias Felber, Austen Worth, et al.
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