199 results on '"Tucci, Francesca"'
Search Results
2. Long-term and real-world safety and efficacy of retroviral gene therapy for adenosine deaminase deficiency
3. A Novel Assay in Whole Blood Demonstrates Restoration of Mitochondrial Activity in Phagocytes After Successful HSCT in Hyperinflamed X-Linked Chronic Granulomatous Disease
4. A systematic review and meta-analysis of gene therapy with hematopoietic stem and progenitor cells for monogenic disorders
5. 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
6. Design of a Multi-Center Randomized Active Controlled Phase 3 Clinical Trial (HURCULES) Evaluating the Safety and Efficacy of OTL-203 in Patients with MPS-IH Versus Standard of Care with Allogeneic Hematopoietic Stem Cell Transplantation
7. Design of a multi-center randomized active controlled phase 3 clinical trial (HURCULES) evaluating the safety and efficacy of OTL-203 in patients with MPS IH versus standard of care with allogeneic hematopoietic stem cell transplantation
8. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome
9. Early skeletal outcomes after hematopoietic stem and progenitor cell gene therapy for Hurler syndrome.
10. Treatment with rapamycin can restore regulatory T-cell function in IPEX patients
11. Bone marrow harvesting from paediatric patients undergoing haematopoietic stem cell gene therapy
12. Current and Future Perspective in Hematopoietic Stem Progenitor Cell-gene Therapy for Inborn Errors of Metabolism
13. S250: UNVEILING THE BIOLOGICAL ROLE OF PERIPHERAL BLOOD HUMAN CIRCULATING HEMATOPOIETIC STEM AND PROGENITOR CELLS
14. 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
15. Hemophagocytic inflammatory syndrome in ADA-SCID: report of two cases and literature review
16. Outcome of BCG Vaccination in ADA-SCID Patients: A 12-Patient Series
17. P037: Design of a multi-center randomized phase 3 clinical trial (HURCULES) evaluating OTL-203 in MPS-IH vs allogeneic hematopoietic stem cell transplantation
18. «Ich kann Nietzsche nicht böse sein, weil er mir meine Deutschen verdorben hat…». Thomas Mann lettore della Geburt der Tragödie
19. Adaptive Routes of Hematopoietic Stem Cell Differentiation to Disease Conditions and Age in Gene Therapy Patients
20. Unveiling the Biological Role of Peripheral Blood Human Circulating Hematopoietic Stem and Progenitor Cells
21. Case Report: Consistent disease manifestations with a staggered time course in two identical twins affected by adenosine deaminase 2 deficiency
22. Early response does not predict outcome in children and adolescents with chronic myeloid leukaemia treated with high‐dose imatinib
23. Evidence of treatment benefits in patients with MPSI-Hurler in long-term follow up using a new MRI scoring system
24. First-in-human phase I/II clinical trial of hematopoietic stem and progenitor cell gene therapy for Hurler syndrome: Favorable safety profile and extensive metabolic correction
25. Wiskott–Aldrich syndrome: Oral findings and microbiota in children and review of the literature
26. Nathan und die Ringparabel im Kontext antisemitischer Lessing-Darstellungen
27. Evidence of treatment benefits in patients with MPSI-Hurler in long-term follow up using a new MRI scoring system
28. 'Von den lateinischen Trauerspielen welche unter dem Namen des Seneca bekannt sind' Lessings Auseinandersetzung mit Seneca
29. Evidence of Treatment Benefits in Patients with Mucopolysaccharidosis Type I-Hurler in Long-term Follow-up Using a New Magnetic Resonance Imaging Scoring System
30. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
31. Diagnosis of Respiratory Syncytial Virus (RSV) infection in children by Respiratory Panel utilized during the COVID-19 pandemic.
32. «Per lo più senza desideri e un poco infelici». Ritratto della madre perduta in Wunschloses Unglück di Peter Handke
33. Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated BCGitis
34. Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients
35. Peripheral blood stem and progenitor cell collection in pediatric candidates for ex vivo gene therapy: a 10-year series
36. Long-term results of high-dose imatinib in children and adolescents with chronic myeloid leukaemia in chronic phase: the Italian experience
37. Gene therapy with hematopoietic stem and progenitor cell for monogenic disorders: a systematic review and meta-analysis
38. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome
39. Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200
40. Ex vivo hematopoietic stem cell gene therapy for mucopolysaccharidosis type I (Hurler syndrome)
41. Update on Clinical Ex Vivo Hematopoietic Stem Cell Gene Therapy for Inherited Monogenic Diseases
42. Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report
43. A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation.
44. Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients
45. Extensive Metabolic Correction of Hurler Disease By Hematopoietic Stem Cell-Based Gene Therapy: Preliminary Results from a Phase I/II Trial
46. Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Wiskott-Aldrich Syndrome (WAS): Up to 8 Years of Follow up in 17 Subjects Treated Since 2010
47. Io scrivo ciò che vedo. Einmal di Wim Wenders tra immagini autocelebrative e priorità del racconto
48. Successful Treatment With Ledipasvir/Sofosbuvir in an Infant With Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency With HCV Allowed Gene Therapy with Strimvelis
49. Disease evolution and response to rapamycin in activated phosphoinositide 3-kinase δ syndrome : The European society for immunodeficiencies-activated phosphoinositide 3-kinase δ syndrome registry
50. «Vivere da soldato senza essere tale». Gli scritti di guerra di Thomas Mann
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