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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.The New England Journal of Medicine|January 31, 2009
Gene therapy for immunodeficiency due to adenosine deaminase deficiencyAlessandro Aiuti, Federica Cattaneo, Stefania Galimberti, et al.Blood|April 14, 2026
T cells dressed up with a dual HLA-restricted TCR targeting cathepsin G drive effective AML eradicationFrancesca Marzuttini, Alessia Potenza, Ludovica Celli, 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.Science (New York, N.Y.)|July 13, 2013
Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndromeAlessandro Aiuti, Luca Biasco, Samantha Scaramuzza, et al.The Lancet. Haematology|April 15, 2019
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 studyFrancesca Ferrua, Maria Pia Cicalese, Stefania Galimberti, et al.Pageof 4