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

2. Correction: Rare predicted loss-of-function variants of type I IFN immunity genes are associated with life-threatening COVID-19

4. Rare predicted loss-of-function variants of type I IFN immunity genes are associated with life-threatening COVID-19

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

6. A GLB1 transgene with enhanced therapeutic potential for the preclinical development of ex-vivo gene therapy to treat mucopolysaccharidosis type IVB

8. Lack of association between classical HLA genes and asymptomatic SARS-CoV-2 infection

9. Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

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

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

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

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

14. Autoantibodies neutralizing type I IFNs are present in ~4% of uninfected individuals over 70 years old and account for ~20% of COVID-19 deaths

15. Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity

16. Coronavirus disease 2019 in patients with inborn errors of immunity: An international study

18. Gene therapy for Wiskott-Aldrich syndrome: History, new vectors, future directions

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

21. Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress

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

25. Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency.

26. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study

28. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis

30. 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

32. Autonomous role of Wiskott-Aldrich syndrome platelet deficiency in inducing autoimmunity and inflammation

33. Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study.

34. From Your Nose to Your Toes: A Review of Severe Acute Respiratory Syndrome Coronavirus 2 Pandemic‒Associated Pernio

35. 16 Bone marrow damage in patients with Adenosine Deaminase 2 Deficiency

36. Initial presenting manifestations in 16,486 patients with inborn errors of immunity include infections and noninfectious manifestations

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

38. Primary immunodeficiency diseases: Genomic approaches delineate heterogeneous Mendelian disorders

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

40. Retrieval of vector integration sites from cell-free DNA

41. Wiskott-Aldrich syndrome: a study of 577 patients defines the genotype as a biomarker for disease severity and survival

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

43. Wiskott-Aldrich Syndrome: A study on 577 patients defining the genotype as a predictive biomarker for disease severity

46. Treatment with rapamycin can restore regulatory T-cell function in IPEX patients

47. 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

48. Atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy) preserves cognitive and motor development in early-onset metachromatic leukodystrophy with up to 12 years follow-up

49. Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD)

50. Oncogene-induced senescence in hematopoietic progenitors features myeloid restricted hematopoiesis, chronic inflammation and histiocytosis

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