Biblio

Author Title [ Type(Asc)] Year
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Journal Article
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.
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.
Hernández RJofra, Calabria A, Sanvito F, De Mattia F, Farinelli G, Scala S, Visigalli I, Carriglio N, De Simone M, Vezzoli M, et al. Hematopoietic Tumors in a Mouse Model of X-linked Chronic Granulomatous Disease after Lentiviral Vector-Mediated Gene Therapy. Mol Ther. 2020.
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.
Consiglieri G, Tucci F, De Pellegrin M, Guerrini B, Cattoni A, Risca G, Scarparo S, Sarzana M, Pontesilli S, Mellone R, et al. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome. Sci Transl Med. 2024;16(745):eadi8214.
Cesana D, Cicalese MPia, Calabria A, Merli P, Caruso R, Volpin M, Rudilosso L, Migliavacca M, Barzaghi F, Fossati C, et al. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID. Nat Commun. 2024;15(1):3662.