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Blood|July 28, 2009
ADA-deficient SCID is associated with a specific microenvironment and bone phenotype characterized by RANKL/OPG imbalance and osteoblast insufficiencyAisha V Sauer, Emanuela Mrak, Raisa Jofra Hernandez, et al.
Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation|December 8, 2009
Unpredictability of intravenous busulfan pharmacokinetics in children undergoing hematopoietic stem cell transplantation for advanced beta thalassemia: limited toxicity with a dose-adjustment policyRobert Chiesa, Barbara Cappelli, Roberto Crocchiolo, et al.
Science (New York, N.Y.)|June 29, 2002
Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioningAlessandro Aiuti, Shimon Slavin, Memet Aker, et al.
The Journal of Allergy and Clinical Immunology|March 21, 2015
B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndromeMaria Carmina Castiello, Samantha Scaramuzza, Francesca Pala, et al.
Journal of Translational Medicine|July 14, 2007
Molecular purging of multiple myeloma cells by ex-vivo culture and retroviral transduction of mobilized-blood CD34+ cellsSara Deola, Samantha Scaramuzza, Roberto Sciarretta Birolo, et al.
EMBO Molecular Medicine|July 29, 2010
Correction of beta-thalassemia major by gene transfer in haematopoietic progenitors of pediatric patientsEmanuela Anna Roselli, Riccardo Mezzadra, Marta Claudia Frittoli, et al.
Blood|September 10, 2009
Wild-type FOXP3 is selectively active in CD4+CD25(hi) regulatory T cells of healthy female carriers of different FOXP3 mutationsSara Di Nunzio, Massimiliano Cecconi, Laura Passerini, et al.
Blood|November 22, 2012
A novel function for FOXP3 in humans: intrinsic regulation of conventional T cellsAlicia N McMurchy, Jana Gillies, Maria Concetta Gizzi, et al.
The Journal of Experimental Medicine|March 25, 2009
The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and functionMichela Locci, Elena Draghici, Francesco Marangoni, et al.
Frontiers in Immunology|February 20, 2018
First Occurrence of Plasmablastic Lymphoma in Adenosine Deaminase-Deficient Severe Combined Immunodeficiency Disease Patient and Review of the LiteratureMaddalena Migliavacca, Andrea Assanelli, Maurilio Ponzoni, et al.
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