Biblio

Author Title [ Type(Desc)] Year
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Journal Article
Quaranta P, Basso-Ricci L, Hernández RJofra Jofr, Pacini G, Naldini MMaria, Barcella M, Seffin L, Pais G, Spinozzi G, Benedicenti F, et al. Circulating Hematopoietic Stem/Progenitor Cells subsets contribute to human hematopoietic homeostasis. Blood. 2024.
Gentner B, Tucci F, Galimberti S, Fumagalli F, De Pellegrin M, Silvani P, Camesasca C, Pontesilli S, Darin S, Ciotti F, et al. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome. N Engl J Med. 2021;385(21):1929-1940.
Fratini ESophia, Migliavacca M, Barzaghi F, Fossati C, Giannelli S, Monti I, Casiraghi M, Ferrua F, Recupero S, Consiglieri G, et al. Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review. Front Immunol. 2023;14:1187959.
Marktel S, Scaramuzza S, Cicalese MPia, Giglio F, Galimberti S, Lidonnici MRosa, Calbi V, Assanelli A, Bernardo MEster, Rossi C, et al. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia. Nat Med. 2019.
Schoenmakers DH, Mochel F, Adang LA, Boelens J-J, Calbi V, Eklund EA, Grønborg SW, Fumagalli F, Groeschel S, Lindemans C, et al. Inventory of current practices regarding hematopoietic stem cell transplantation in metachromatic leukodystrophy in Europe and neighboring countries. Orphanet J Rare Dis. 2024;19(1):46.
Ferrua F, Cicalese MPia, Galimberti S, Giannelli S, Dionisio F, Barzaghi F, Migliavacca M, Bernardo MEster, Calbi V, Assanelli AAngelo, et al. Lentiviral haemopoietic stem/progenitor cell gene therapy for treatment of Wiskott-Aldrich syndrome: interim results of a non-randomised, open-label, phase 1/2 clinical study. Lancet Haematol. 2019.
Laugwitz L, Schoenmakers DH, Adang LA, Beck-Woedl S, Bergner C, Bernard G, Bley A, Boyer A, Calbi V, Dekker H, et al. Newborn screening in metachromatic leukodystrophy - European consensus-based recommendations on clinical management. Eur J Paediatr Neurol. 2024;49:141-154.