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Nature Medicine|March 27, 2019
Highly efficient therapeutic gene editing of human hematopoietic stem cellsYuxuan Wu, Jing Zeng, Benjamin P Roscoe, et al.
Eclinicalmedicine|June 23, 2025
Long-term outcome in Wiskott-Aldrich syndrome and X-linked thrombocytopenia patients: an observational -prospective multi-center study of the Italian Primary Immune Deficiency Network (IPINET)Annarosa Soresina, Roberto Rondelli, Lucia Dora Notarangelo, et al.
Bone Marrow Transplantation|May 19, 2026
The impact of pre-transplant extramedullary disease on the outcome of allogeneic hematopoietic cell transplantation for acute myeloid leukaemia in children- on behalf of PDWP/EBMTJuliana Silva, Jacques-Emmanuel Galimard, Jean-Hugues Dalle, et al.
Bone Marrow Transplantation|October 29, 2025
Comparison of outcomes of BuCy versus BuCyMel as preparation for allogeneic HSCT in infants with acute myeloid leukemia in first complete remission: a multicenter study from the EBMT PDWPAndre Manfred Willasch, Claire Horgan, Christina Peters, et al.
Transplantation and Cellular Therapy|March 7, 2024
Umbilical Cord Blood Transplantation for Fanconi Anemia With a Special Focus on Late Complications: a Study on Behalf of Eurocord and SAAWP-EBMTHanadi Rafii, Fernanda Volt, Marc Bierings, et al.
Lancet (London, England)|June 13, 2016
Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trialMaria Sessa, Laura Lorioli, Francesca Fumagalli, et al.
Bone Marrow Transplantation|June 1, 2019
Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapyFrancesca Tucci, Marta Frittoli, Federica Barzaghi, et al.
Blood|January 12, 2024
CD371-positive pediatric B-cell acute lymphoblastic leukemia: propensity to lineage switch and slow early response to treatmentBarbara Buldini, Elena Varotto, Margarita Maurer-Granofszky, et al.
Human Genetics|June 29, 2021
IFIH1 loss-of-function variants contribute to very early-onset inflammatory bowel diseaseMara Cananzi, Elizabeth Wohler, Antonio Marzollo, et al.
Blood|April 26, 2024
Outcomes of HLA-mismatched HSCT with TCRαβ/CD19 depletion or post-HSCT cyclophosphamide for inborn errors of immunitySu Han Lum, Michael H Albert, Patrick Gilbert, et al.
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