Biblio
Treosulfan-based conditioning regimen for children and adolescents with hemophagocytic lymphohistiocytosis. Haematologica. 2013.
Treosulfan vs busulfan conditioning for allogeneic bmt in children with nonmalignant disease: a randomized phase 2 trial. Bone Marrow Transplant. 2023.
Treatment with Rapamycin Can Restore Regulatory T Cell Function in IPEX Patients. J Allergy Clin Immunol. 2019.
Successful hematopoietic stem cell transplantation in a 4-1BB deficient patient with EBV-induced lymphoproliferation. Clin Immunol. 2020:108639.
Regulation and Function of Lentiviral Vector-Mediated TCIRG1 Expression in Osteoclasts from Patients with Infantile Malignant Osteopetrosis: Implications for Gene Therapy. Calcif Tissue Int. 2016.
. Publisher Correction: Clinical evolution, genetic landscape and trajectories of clonal hematopoiesis in SAMD9/SAMD9L syndromes. Nat Med. 2021.
Persisting enteropathy and disturbed adaptive mucosal immunity due to MHC class II deficiency. Clin Immunol. 2019.
Osteopetrosis: genetics, treatment and new insights into osteoclast function. Nat Rev Endocrinol. 2013.
. [Newborn screening for severe combined immunodeficiencies (SCID) in Germany]. Bundesgesundheitsblatt Gesundheitsforschung Gesundheitsschutz. 2023.
. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency. J Allergy Clin Immunol. 2015.
International retrospective study of allogeneic hematopoietic cell transplantation for activated PI3K-delta syndrome. J Allergy Clin Immunol. 2021.
Favorable outcomes of hematopoietic stem cell transplantation in children and adolescents with Diamond-Blackfan anemia. Blood Adv. 2020;4(8):1760-1769.
Cutaneous barrier leakage and gut inflammation drive skin disease in Omenn Syndrome. J Allergy Clin Immunol. 2020.
Clearance of hematological malignancies by allogeneic cytokine-induced killer cell or donor lymphocyte infusions. Biol Blood Marrow Transplant. 2019.