Biblio

Author Title [ Type(Desc)] Year
Filters: Author is Ferrari, Giuliana  [Clear All Filters]
Journal Article
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.
Lidonnici MRosa, Scaramuzza S, Ferrari G. Gene therapy for Hemoglobinopathies. Hum Gene Ther. 2023.
Aprile A, Gulino A, Storto M, Villa I, Beretta S, Merelli I, Rubinacci A, Ponzoni M, Marktel S, Tripodo C, et al. Hematopoietic stem cell function in β-thalassemia is impaired and is rescued by targeting the bone marrow niche. Blood. 2020.
Aprile A, Raggi L, Bolamperti S, Villa I, Storto M, Morello G, Marktel S, Tripodo C, Cappellini MDomenica, Motta I, et al. Inhibition of FGF23 is a therapeutic strategy to target hematopoietic stem cell niche defects in β-thalassemia. Sci Transl Med. 2023;15(698):eabq3679.
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.
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.
Valeri E, Unali G, Piras F, Abou-Alezz M, Pais G, Benedicenti F, Lidonnici MRosa, Cuccovillo I, Castiglioni I, Arévalo S, et al. Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cells. Mol Ther. 2023.