Biblio

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Journal Article
Vallée TC, Glasmacher JS, Buchner H, Arkwright PD, Behrends U, Bondarenko A, Browning MJ, Buchbinder DK, Cattoni A, Chernyshova L, et al. Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity. Blood. 2024.
Lum SHan, Greener S, Perez-Heras I, Drozdov D, Payne RPamela, Watson H, Carruthers K, January R, Nademi Z, Owens S, et al. T-replete HLA-matched Grafts vs T-depleted HLA-mismatched Grafts in Inborn Errors of Immunity. Blood Adv. 2021.
Tsilifis C, Lum SHan, Nademi Z, Hambleton S, Flood TJ, Williams EJ, Owens S, Abinun M, Cant AJ, Slatter MA, et al. TCRαβ-Depleted Haploidentical Grafts Are a Safe Alternative to HLA-Matched Unrelated Donor Stem Cell Transplants for Infants with Severe Combined Immunodeficiency. J Clin Immunol. 2022.
Tsilifis C, Moreira D, Marques L, Neves E, Slatter MA, Gennery AR. Stem cell transplantation as treatment for major histocompatibility class I deficiency. Clin Immunol. 2021:108801.
Burns H, Collins A, Marsden P, Flood TJ, Slatter MA, Booth C, Xu-Bayford J, Leahy TRonan. Severe Combined Immunodeficiency (SCID)-the Irish Experience. J Clin Immunol. 2021.
Mehta P, Tsilifis C, Lum SHan, Slatter MA, Hambleton S, Owens S, Williams E, Flood T, Gennery AR, Nademi Z. Outcome of Second Allogeneic HSCT for Patients with Inborn Errors of Immunity: Retrospective Study of 20 Years' Experience. J Clin Immunol. 2023.
Radwan N, Nademi Z, Lum SHan, Flood T, Abinun M, Owens S, Williams E, Gennery AR, Hambleton S, Slatter MA. Outcome of Hematopoietic Stem Cell Transplantation in patients with Mendelian Susceptibility to Mycobacterial Diseases. J Clin Immunol. 2021.
Wehr C, Gennery AR, Lindemans C, Schulz A, Hoenig M, Marks R, Recher M, Gruhn B, Holbro A, Heijnen I, et al. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol. 2015.
Fischer M, Olbrich P, Hadjadj J, Aumann V, Bakhtiar S, Barlogis V, von Bismarck P, Bloomfield MPhDMarké, Booth C, Buddingh EP, et al. JAK-inhibitor treatment for inborn errors of JAK/STAT signaling: An ESID and EBMT IEWP retrospective study. J Allergy Clin Immunol. 2023.
Schuetz C, Gerke J, Ege MJohannes, Walter JEszter, Kusters M, Worth AJJ, Kanakry JA, Dimitrova D, Wolska-Kusnierz B, Chen K, et al. Hypomorphic RAG deficiency: impact of disease burden on survival and thymic recovery argues for early diagnosis and HSCT. Blood. 2022.
Perez-Heras I, Tsilifis C, Slatter MA, Brynjólfsson SF, Haraldsson Á, Gennery AR. HSCT in two brothers with CGD arising from mutations in CYBC1 corrects the defect in neutrophil function. Clin Immunol. 2021:108799.
Albert MH, Slatter MA, Gennery AR, Güngör T, Bakunina K, Markovitch B, Hazelaar S, Sirait T, Courteille V, Aiuti A, et al. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT inborn errors working party analysis. Blood. 2022.
Albert MH, Sirait T, Eikema D-J, Bakunina K, Wehr C, Suarez F, Fox MLaura, Mahlaoui N, Gennery AR, Lankester AC, et al. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity, an EBMT IEWP study. Blood. 2022.
Abinun M, Slatter MA. Haematopoietic stem cell transplantation in paediatric rheumatic disease. Curr Opin Rheumatol. 2021.
van der Made CI, Kersten S, Chorin O, Engelhardt KR, Ramakrishnan G, Griffin H, van der Loeff ISchim, Venselaar H, Rothschild ARaas, Segev M, et al. Expanding the PRAAS spectrum: De novo mutations of immunoproteasome subunit β-type 10 in six infants with SCID-Omenn syndrome. Am J Hum Genet. 2024.
Le Voyer T, Parent AV, Liu X, Cederholm A, Gervais A, Rosain J, Nguyen T, Lorenzo MPerez, Rackaityte E, Rinchai D, et al. Autoantibodies against type I IFNs in humans with alternative NF-κB pathway deficiency. Nature. 2023.
Tsilifis C, Torppa T, Williams EJ, Albert MH, Hauck F, Soncini E, Kang E, Malech H, Schuetz C, von Bernuth H, et al. Allogeneic HSCT for Symptomatic Female X-linked Chronic Granulomatous Disease Carriers. J Clin Immunol. 2023.
Slatter MA, Gennery AR. Advances in the treatment of severe combined immunodeficiency. Clin Immunol. 2022:109084.