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The Journal of Clinical Investigation|March 5, 2019
Bone marrow stromal cells from β-thalassemia patients have impaired hematopoietic supportive capacityStefania Crippa, Valeria Rossella, Annamaria Aprile, et al.Molecular Therapy. Methods & Clinical Development|October 16, 2018
Multiple Integrated Non-clinical Studies Predict the Safety of Lentivirus-Mediated Gene Therapy for β-ThalassemiaMaria Rosa Lidonnici, Ylenia Paleari, Francesca Tiboni, et al.Science Translational Medicine|November 26, 2025
Base editing of β<sup>0</sup>-thalassemia mutations as a therapeutic strategy for severe β-hemoglobinopathiesGiulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, et al.Molecular Therapy : the Journal of the American Society of Gene Therapy|November 22, 2023
Removal of innate immune barriers allows efficient transduction of quiescent human hematopoietic stem cellsErika Valeri, Giulia Unali, Francesco Piras, et al.Blood|June 20, 2020
Correcting β-thalassemia by combined therapies that restrict iron and modulate erythropoietin activityCarla Casu, Mariateresa Pettinato, Alison Liu, et al.Blood|January 24, 2022
Unique molecular and functional features of extramedullary hematopoietic stem and progenitor cell reservoirs in humansNicole Mende, Hugo P Bastos, Antonella Santoro, et al.Nature|October 23, 2024
Long-term lineage commitment in haematopoietic stem cell gene therapyAndrea Calabria, Giulio Spinozzi, Daniela Cesana, et al.Nature Communications|July 1, 2025
Imbalanced TGFβ signalling and autophagy drive erythroid priming of hematopoietic stem cells in β-thalassemiaMaria Rosa Lidonnici, Giulia Chianella, Nicole Mende, et al.Nature Medicine|January 22, 2019
Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemiaSarah Marktel, Samantha Scaramuzza, Maria Pia Cicalese, et al.Pageof 6