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Frontiers in Immunology|July 12, 2023
Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature reviewElena Sophia Fratini, Maddalena Migliavacca, Federica Barzaghi, et al.
Nature|October 23, 2024
Long-term lineage commitment in haematopoietic stem cell gene therapyAndrea Calabria, Giulio Spinozzi, Daniela Cesana, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|September 29, 2025
Non-neurological, non-skeletal outcomes after hematopoietic stem and progenitor cell-gene therapy (OTL-203) for Hurler syndromeFrancesca Tucci, M Luz Uria Oficialdegui, Giulia Consiglieri, 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.
Frontiers in Immunology|December 17, 2020
Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case ReportSabina Cenciarelli, Valeria Calbi, Federica Barzaghi, et al.
Disease Models & Mechanisms|March 5, 2026
Development and characterization of a model of mucopolysaccharidosis type IVA for evaluating therapies targeting bone diseaseMargherita Berti, Selene Ceriotti, Ludovica Santi, et al.
Frontiers in Immunology|October 17, 2022
Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiencyFederica Barzaghi, Maria Pia Cicalese, Matteo Zoccolillo, et al.
Nature Communications|April 30, 2024
A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCIDDaniela Cesana, Maria Pia Cicalese, Andrea Calabria, et al.
Science Translational Medicine|May 1, 2024
Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndromeGiulia Consiglieri, Francesca Tucci, Maurizio De Pellegrin, et al.
Plos Genetics|March 9, 2011
Meta-analysis of genome-wide association studies in celiac disease and rheumatoid arthritis identifies fourteen non-HLA shared lociAlexandra Zhernakova, Eli A Stahl, Gosia Trynka, et al.
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