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Cell Stem Cell|October 7, 2022
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cellsSamuele Ferrari, Aurelien Jacob, Daniela Cesana, et al.
Human Mutation|December 3, 2009
Molecular and clinical heterogeneity in CLCN7-dependent osteopetrosis: report of 20 novel mutationsAlessandra Pangrazio, Michael Pusch, Elena Caldana, et al.
Frontiers in Immunology|April 17, 2024
Autoimmunity in thymic epithelial tumors: a not yet clarified pathologic paradigm associated with several unmet clinical needsMatteo Perrino, Emanuele Voulaz, Simone Balin, et al.
Nature Medicine|May 19, 2009
Impaired gastric acidification negatively affects calcium homeostasis and bone massThorsten Schinke, Arndt F Schilling, Anke Baranowsky, et al.
Nature|May 28, 2025
In vivo haemopoietic stem cell gene therapy enabled by postnatal traffickingMichela Milani, Anna Fabiano, Marta Perez-Rodriguez, et al.
Molecular Therapy. Methods & Clinical Development|April 24, 2020
Successful Preclinical Development of Gene Therapy for Recombinase-Activating Gene-1-Deficient SCIDLaura Garcia-Perez, Marja van Eggermond, Lieke van Roon, et al.
Frontiers in Immunology|July 2, 2021
Thymic Epithelial Cell Alterations and Defective Thymopoiesis Lead to Central and Peripheral Tolerance Perturbation in MHCII DeficiencyFrancesca Ferrua, Ileana Bortolomai, Elena Fontana, et al.
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