Biblio

Author Title Type [ Year(Asc)]
Filters: Author is Scaramuzza, Samantha  [Clear All Filters]
2023
Lidonnici MRosa, Scaramuzza S, Ferrari G. Gene therapy for Hemoglobinopathies. Hum Gene Ther. 2023.
Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, et al. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies. Mol Ther Nucleic Acids. 2023;32:229-246.
2022
Hardouin G, Antoniou P, Martinucci P, Felix T, Manceau S, Joseph L, Masson C, Scaramuzza S, Ferrari G, Cavazzana M, et al. Adenine base editor-mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation. Blood. 2022.
2019
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.
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.
2015
Neri T, Muggeo S, Paulis M, Caldana MElena, Crisafulli L, Strina D, Focarelli MLuisa, Faggioli F, Recordati C, Scaramuzza S, et al. Targeted Gene Correction in Osteopetrotic-Induced Pluripotent Stem Cells for the Generation of Functional Osteoclasts. Stem Cell Reports. 2015.