390 results on '"Cicalese Maria Pia"'
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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
10. A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation
11. Harnessing Type I IFN Immunity Against SARS-CoV-2 with Early Administration of IFN-β
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
21. A Prevalent CXCR3+ Phenotype of Circulating Follicular Helper T Cells Indicates Humoral Dysregulation in Children with Down Syndrome
22. Gene Therapy for Primary Immunodeficiencies
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
27. T regulatory cell therapy in preclinical and clinical pancreatic islet transplantation
28. T-cell defects in patients with ARPC1B germline mutations account for combined immunodeficiency
29. Gene Therapy for Adenosine Deaminase Deficiency: A Comprehensive Evaluation of Short- and Medium-Term Safety
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
32. Correction to: A Case of Two Adult Brothers with Wiskott‑Aldrich Syndrome, One Treated with Gene Therapy and One with HLA‑Identical Hematopoietic Stem Cell Transplantation
33. Pediatric cancer care management during the COVID-19 pandemic: a review of the literature and a single-centre real-life experience of an Italian pediatric oncology unit
34. The Inborn Errors of Immunity—Virtual Consultation System Platform in Service for the Italian Primary Immunodeficiency Network: Results from the Validation Phase.
35. Dynamics of genetically engineered hematopoietic stem and progenitor cells after autologous transplantation in humans
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
43. Inflammation Converts Human Mesoangioblasts Into Targets of Alloreactive Immune Responses: Implications for Allogeneic Cell Therapy of DMD
44. Gene therapy in rare diseases: the benefits and challenges of developing a patient-centric registry for Strimvelis in ADA-SCID
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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