318 results on '"Aiuti, Alessandro"'
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2. Circulating hematopoietic stem/progenitor cell subsets contribute to human hematopoietic homeostasis
3. 16 Bone marrow damage in patients with Adenosine Deaminase 2 Deficiency
4. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
5. 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
6. Lentiviral hematopoietic stem cell gene therapy (atidarsagene autotemcel) for late juvenile metachromatic leukodystrophy (MLD)
7. Molecular and phenotypic blueprint of the hematopoietic compartment reveals proliferation stress as a driver of age-associated human stem cell dysfunctions
8. Realizing the Potential of Gene Therapies for Rare and Ultra-Rare Inherited Diseases
9. 115 The mannose-binding lectin (MBL) in MIS-C: relationship between MBL genotype, levels, and functional activity against SARS-CoV-2
10. S250: UNVEILING THE BIOLOGICAL ROLE OF PERIPHERAL BLOOD HUMAN CIRCULATING HEMATOPOIETIC STEM AND PROGENITOR CELLS
11. P1392: INTERIM ANALYSIS OF FIRST IN HUMAN PHASE I-II CLINICAL TRIAL OF EX-VIVO GENE THERAPY FOR HURLER SYNDROME: AN UPDATE AT 3 YEAR FOLLOW-UP
12. Newborn screening for severe combined immunodeficiency: changing the landscape of post-transplantation survival
13. Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review
14. Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series
15. Autoantibodies neutralizing type I IFNs underlie West Nile virus encephalitis in ∼40% of patients
16. Activated phosphoinositide 3-kinase δ syndrome: Update from the ESID Registry and comparison with other autoimmune-lymphoproliferative inborn errors of immunity
17. Biological Properties of HSC: Scientific Basis for HSCT
18. Constitutive IL-1RA production by modified immune cells protects against IL-1–mediated inflammatory disorders
19. Hematopoietic reconstitution dynamics of mobilized- and bone marrow-derived human hematopoietic stem cells after gene therapy
20. Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
21. Overhydrated hereditary stomatocytosis: A rare cause of familiar persistent macrocytosis due to SLC4A1 variants
22. Preserving Ambulation in a Gene Therapy-Treated Girl Affected by Metachromatic Leukodystrophy: A Case Report
23. Inherited and acquired errors of type I interferon immunity govern susceptibility to COVID-19 and multisystem inflammatory syndrome in children
24. Towards improved yet regulated gene therapy for X-CGD
25. Gene Therapy for Primary Immunodeficiencies
26. Blood sulfatides as disease biomarker for metachromatic leukodystrophy: Disease characterization, early diagnosis, and response to treatment
27. Atidarsagene Autotemcel, a European Post-Regulatory Approval Model for Delivery of Autologous Hematopoietic Stem Cell Gene Therapy Products Via a Network of Qualified Treatment Centers (QTCs)
28. Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use
29. Atidarsagene autotemcel, a European post-regulatory approval model for delivery of autologous hematopoietic stem cell gene therapy products via a network of qualified treatment centers (QTCs)
30. A Brazilian patient with late infantile metachromatic leukodystrophy treated with lentiviral hematopoietic stem-cell gene therapy: A report from prenatal diagnosis to early treatment
31. Long-term clinical outcomes of atidarsagene autotemcel (autologous hematopoietic stem cell gene therapy [HSC-GT] for metachromatic leukodystrophy) with up to 11 years follow-up
32. Updated Management Guidelines for Adenosine Deaminase Deficiency
33. Genetic determinants of type 1 diabetes in individuals with weak evidence of islet autoimmunity at disease onset
34. Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs
35. Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients
36. Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells
37. Hematopoietic stem cell transplantation for adolescents and adults with inborn errors of immunity: an EBMT IEWP study
38. Mesenchymal stromal cells improve the transplantation outcome of CRISPR-Cas9 gene-edited human HSPCs
39. Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency
40. Prikaz primera prvega uspešnega genskega zdravljenja slovenskega bolnika z mukopolisaharidozo tipa I v tujini
41. Cellular and transcriptional dynamics of human neutrophils at steady state and upon stress
42. Natural history of type 1 diabetes on an immunodysregulatory background with genetic alteration in B-cell activating factor receptor: A case report
43. Ensuring a future for gene therapy for rare diseases
44. A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease
45. Ex Vivo and In Vivo Gene Therapy for Mucopolysaccharidoses: State of the Art
46. SARS‐CoV‐2 infection and treatment in a cohort of patients with inborn errors of immunity
47. Radiosensitivity in patients affected by ARPC1B deficiency: a new disease trait?
48. Follicular helper T cell signature of replicative exhaustion, apoptosis, and senescence in common variable immunodeficiency
49. The Impact of SARS-CoV-2 Infection in Patients with Inborn Errors of Immunity: the Experience of the Italian Primary Immunodeficiencies Network (IPINet)
50. Hematopoietic stem cell transplantation for Wiskott-Aldrich syndrome: an EBMT Inborn Errors Working Party analysis
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