Showing results (121-130 of 169) with videos related to
Sort By:
Pageof 17
Molecular Therapy. Methods & Clinical Development|September 11, 2023
Scalable GMP-compliant gene correction of CD4+ T cells with IDLV template functionally validated in vitro and in vivoClaudia Asperti, Daniele Canarutto, Simona Porcellini, et al.The Journal of Clinical Investigation|April 4, 2007
Involvement of PLEKHM1 in osteoclastic vesicular transport and osteopetrosis in incisors absent rats and humansLiesbeth Van Wesenbeeck, Paul R Odgren, Fraser P Coxon, et al.Nature Biotechnology|June 1, 2026
Selection of human hematopoietic stem cells bearing the intended functional edit by transient AND-gate reportersDaniele Canarutto, Martina Fiumara, Vigneshwaran Venkatesan, et al.Science Translational Medicine|October 13, 2017
Preclinical modeling highlights the therapeutic potential of hematopoietic stem cell gene editing for correction of SCID-X1Giulia Schiroli, Samuele Ferrari, Anthony Conway, 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.The Journal of Experimental Medicine|June 16, 2010
Homeostatic expansion of autoreactive immunoglobulin-secreting cells in the Rag2 mouse model of Omenn syndromeBarbara Cassani, Pietro Luigi Poliani, Veronica Marrella, et al.Journal of Bone and Mineral Research : the Official Journal of the American Society for Bone and Mineral Research|October 31, 2003
Chloride channel ClCN7 mutations are responsible for severe recessive, dominant, and intermediate osteopetrosisAnnalisa Frattini, Alessandra Pangrazio, Lucia Susani, et al.Frontiers in Endocrinology|September 24, 2024
Correction of osteopetrosis in the neonate oc/oc murine model after lentiviral vector gene therapy and non-genotoxic conditioningSara Penna, Alessandra Zecchillo, Martina Di Verniere, et al.Frontiers in Immunology|April 6, 2019
Thymic Epithelium Abnormalities in DiGeorge and Down Syndrome Patients Contribute to Dysregulation in T Cell DevelopmentGenni Enza Marcovecchio, Ileana Bortolomai, Francesca Ferrua, et al.The Journal of Allergy and Clinical Immunology|December 16, 2017
Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulationValentina Capo, Maria Carmina Castiello, Elena Fontana, et al.Pageof 17