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Your search keyword '"Gallo, Vera"' showing total 194 results

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

2. 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

3. DiGeorge Syndrome

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

8. Rituximab Unveils Hypogammaglobulinemia and Immunodeficiency in Children with Autoimmune Cytopenia

9. 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

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

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

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

16. P005: Atidarsagene autotemcel (hematopoietic stem cell–gene therapy) preserves cognitive and motor development in metachromatic leukodystrophy with up to 12 years follow-up*

18. Blood sulfatides as disease biomarker for metachromatic leukodystrophy: Disease characterization, early diagnosis, and response to treatment

19. Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in 5 patients treated under nominal compassionate use

20. 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

22. Wiskott–Aldrich syndrome: Oral findings and microbiota in children and review of the literature

23. Emapalumab treatment in an ADA-SCID patient with refractory hemophagocytic lymphohistiocytosis-related graft failure and disseminated BCGitis

24. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

25. Lentiviral Hematopoietic Stem and Progenitor Cell Gene Therapy for Metachromatic Leukodystrophy (MLD): Clinical Outcomes from 38 Patients

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

29. Hematopoietic Stem- and Progenitor-Cell Gene Therapy for Hurler Syndrome

31. Lentiviral haematopoietic stem cell gene therapy for metachromatic leukodystrophy: Results in nine patients treated with a cryopreserved formulation of OTL-200

32. Lentiviral hematopoietic stem and progenitor cell gene therapy provides durable clinical benefit in early-symptomatic early-juvenile metachromatic leukodystrophy

35. Mild SARS-CoV-2 Infection After Gene Therapy in a Child With Wiskott-Aldrich Syndrome: A Case Report

36. In Ataxia-Telangiectasia, Oral Betamethasone Administration Ameliorates Lymphocytes Functionality through Modulation of the IL-7/IL-7Rα Axis Paralleling the Neurological Behavior: A Comparative Report of Two Cases

37. Clinical, Immunological, and Functional Characterization of Six Patients with Very High IgM Levels

38. Lentiviral hematopoietic stem and progenitor cell gene therapy (HSPC-GT) for metachromatic leukodystrophy (MLD): Clinical outcomes from 33 patients

39. A Case of Two Adult Brothers with Wiskott-Aldrich Syndrome, One Treated with Gene Therapy and One with HLA-Identical Hematopoietic Stem Cell Transplantation.

41. Subcutaneous Immunoglobulin Twenty Percent Every Two Weeks in Pediatric Patients with Primary Immunodeficiencies: Subcohort Analysis of the IBIS Study

42. In Ataxia-Telangiectasia, Oral Betamethasone Administration Ameliorates Lymphocytes Functionality through Modulation of the IL-7/IL-7Rα Axis Paralleling the Neurological Behavior: A Comparative Report of Two Cases.

43. NADPH Oxidase Deficiency: A Multisystem Approach

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

45. Corrigendum to: “Vaccination in immunocompromised host: Recommendations of Italian Primary Immunodeficiency Network Centers (IPINET)” [Vaccine 36 (2018) Pages 3541–3542]

49. Intrathecal amphotericin B therapy in a patient with X-linked chronic granulomatous disease and refractory cerebral invasive aspergillosis

50. TLR9 signaling in patients with ectodermal dysplasia and immunodeficiency associated with Nuclear Factor Essential Modulator (NEMO) mutations

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