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1. Exonic knockout and knockin gene editing in hematopoietic stem and progenitor cells rescues RAG1 immunodeficiency

3. Immunopathological signatures in multisystem inflammatory syndrome in children and pediatric COVID-19

4. Auto-antibodies against type I IFNs in patients with life-threatening COVID-19

6. Autoantibodies against type I IFNs in patients with life-threatening COVID-19

7. Neutrophils drive type-I interferon production and autoantibodies in Wiskott-Aldrich syndrome

9. Efficacy of lentivirus-mediated gene therapy in an Omenn syndrome recombination-activating gene 2 mouse model is not hindered by inflammation and immune dysregulation

10. Lentiviral-mediated gene therapy restores B cell tolerance in Whiskott-Aldrich syndrome patients

12. Wiskott-Aldrich Syndrome protein deficiency perturbs the homeostasis of B-cell compartment in humans

14. LENTIVIRAL VECTOR TRANSDUCED CD34+CELLS FOR THE TREATMENT OF WISKOTT-ALDRICH SYNDROME

15. Gene therapy trial with lentiviral vector transduced CD34+cells for the treatment of Wiskott-Aldrich Syndrome

17. B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome

18. B-cell reconstitution after lentiviral vector-mediated gene therapy in patients with Wiskott-Aldrich syndrome

19. Lentivirus-based Gene Therapy of Hematopoietic Stem Cells in Wiskott-Aldrich Syndrome

20. Wiskott-Aldrich Syndrome protein deficiency pertubs the homeostatis of B-cell compartment in humans

21. Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity

23. Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome

24. Hypomorphic Rag1mutations alter the preimmune repertoire at early stages of lymphoid development

31. Perturbations of the T-cell receptor repertoire in response to SARS-CoV-2 in immunocompetent and immunocompromised individuals

32. Inherited ARPC5 mutations cause an actinopathy impairing cell motility and disrupting cytokine signaling

34. Immunopathological signatures in multisystem inflammatory syndrome in children and pediatric COVID-19

35. SASH3 variants cause a novel form of X-linked combined immunodeficiency with immune dysregulation

36. Premature Senescence and Increased Oxidative Stress in the Thymus of Down Syndrome Patients

37. Gene Modification and Three-Dimensional Scaffolds as Novel Tools to Allow the Use of Postnatal Thymic Epithelial Cells for Thymus Regeneration Approaches

38. Lentiviral gene therapy corrects platelet phenotype and function in patients with Wiskott-Aldrich syndrome

39. Efficacy Of Lentivirus-Mediated Gene Therapy In An Omenn Syndrome Recombination-Activating Gene 2 Mouse Model Is Not Hindered By Inflammation And Immune Dysregulation

40. In Vivo Chronic Stimulation Unveils Autoreactive Potential of Wiskott–Aldrich Syndrome Protein-Deficient B Cells

41. The Wiskott-Aldrich syndrome protein is required for iNKT cell maturation and function

42. Wiskott-Aldrich syndrome protein deficiency in natural killer and dendritic cells affects antitumor immunity

43. Preclinical Safety and Efficacy of Human CD34(+) Cells Transduced With Lentiviral Vector for the Treatment of Wiskott-Aldrich Syndrome

44. Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome

45. Dendritic cell functional improvement in a preclinical model of lentiviral-mediated gene therapy for Wiskott-Aldrich syndrome

46. Lentiviral-mediated gene therapy leads to improvement of B-cell functionality in a murine model of Wiskott-Aldrich syndrome

47. Revertant T lymphocytes in a patient with Wiskott-Aldrich syndrome: analysis of function and distribution in lymphoid organs

48. Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome

49. Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott–Aldrich Syndrome in Preclinical Models

50. A hypomorphic R229Q Rag2 mouse mutant recapitulates human Omenn syndrome

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