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390 results on '"Cicalese Maria Pia"'

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1. A case of T-cell acute lymphoblastic leukemia in retroviral gene therapy for ADA-SCID

2. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency

3. Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis

4. Early bone marrow alterations in patients with adenosine deaminase 2 deficiency across disease phenotypes and severities

5. Genetic determinants of type 1 diabetes in individuals with weak evidence of islet autoimmunity at disease onset

6. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

7. A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease

8. The Impact of SARS-CoV-2 Infection in Patients with Inborn Errors of Immunity: the Experience of the Italian Primary Immunodeficiencies Network (IPINet)

9. Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1/2 trial and expanded access

12. Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients

13. Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series

14. Lentiviral correction of enzymatic activity restrains macrophage inflammation in adenosine deaminase 2 deficiency

15. Interleukin-1 blockade in patients with Wiskott-Aldrich syndrome: a retrospective multinational case series

16. COVID-19 in recipients of allogeneic stem cell transplantation: favorable outcome

17. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome

18. Correction to: Hematopoietic Cell Transplantation Cures Adenosine Deaminase 2 Deficiency: Report on 30 Patients

19. Consensus of the Italian Primary Immunodeficiency Network on transition management from pediatric to adult care in patients affected with childhood-onset inborn errors of immunity

20. Reduced PD-1 expression on circulating follicular and conventional FOXP3+ Treg cells in children with new onset type 1 diabetes and autoantibody-positive at-risk children

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

24. 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 study

25. B-cell development and functions and therapeutic options in adenosine deaminase–deficient patients

26. Contributors

28. T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency

30. Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy

31. Intrabone hematopoietic stem cell gene therapy for adult and pediatric patients affected by transfusion-dependent ß-thalassemia

34. The Inborn Errors of Immunity—Virtual Consultation System Platform in Service for the Italian Primary Immunodeficiency Network: Results from the Validation Phase.

36. Use of Defibrotide to help prevent post-transplant endothelial injury in a genetically predisposed infant with metachromatic leukodystrophy undergoing hematopoietic stem cell gene therapy

37. Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review

38. Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series

39. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy

40. Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1/2 trial

41. Update on the safety and efficacy of retroviral gene therapy for immunodeficiency due to adenosine deaminase deficiency

42. Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells

45. Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency

46. Natural history of type 1 diabetes on an immunodysregulatory background with genetic alteration in B-cell activating factor receptor: A case report

47. SARS‐CoV‐2 infection and treatment in a cohort of patients with inborn errors of immunity

48. Human IL2RA null mutation mediates immunodeficiency with lymphoproliferation and autoimmunity

49. Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy

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

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