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Molecular Therapy : the Journal of the American Society of Gene Therapy|March 5, 2009
Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical modelsFrancesco Marangoni, Marita Bosticardo, Sabine Charrier, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 30, 2014
Dual-regulated lentiviral vector for gene therapy of X-linked chronic granulomatosisMaria Chiriaco, Giada Farinelli, Valentina Capo, 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.
The Journal of Allergy and Clinical Immunology|February 18, 2010
Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organsSara Trifari, Samantha Scaramuzza, Marco Catucci, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 29, 2012
Preclinical safety and efficacy of human CD34(+) cells transduced with lentiviral vector for the treatment of Wiskott-Aldrich syndromeSamantha Scaramuzza, Luca Biasco, Anna Ripamonti, et al.
The Journal of Allergy and Clinical Immunology|February 11, 2014
B-cell development and functions and therapeutic options in adenosine deaminase-deficient patientsImmacolata Brigida, Aisha V Sauer, Francesca Ferrua, et al.
Nature Communications|July 6, 2026
Dipeptidyl peptidase 3 sets the threshold for immune activation and survival during experimental bacterial infectionAmanda Facoetti, Luca Lambroia, Elena Fontana, et al.
Nature|October 23, 2024
Long-term lineage commitment in haematopoietic stem cell gene therapyAndrea Calabria, Giulio Spinozzi, Daniela Cesana, et al.
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