111 results on '"Gaspar, Bobby"'
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2. Genetic Disorders of Immune Regulation
3. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation
4. The extended clinical phenotype of 64 patients with dedicator of cytokinesis 8 deficiency
5. Outcome of hematopoietic cell transplantation for DNA double-strand break repair disorders
6. Genetic Disorders of Immune Regulation
7. X-linked Inhibitor of Apoptosis Complicated by Granulomatous Lymphocytic Interstitial Lung Disease (GLILD) and Granulomatous Hepatitis
8. Hematopoietic Stem-Cell Gene Therapy for Cerebral Adrenoleukodystrophy
9. Thymus transplantation for complete DiGeorge syndrome: European experience
10. Tu1723 HEMATOPOIETIC STEM CELL GENE THERAPY AS A TREATMENT FOR SEVERE CROHN'S DISEASE ASSOCIATED WITH PATHOGENIC NOD2 GENETIC VARIANTS
11. Mutations in linker for activation of T cells (LAT) lead to a novel form of severe combined immunodeficiency
12. Long-Term Follow-up Study after Lentiviral Hematopoietic Stem/Progenitor Cell Gene Therapy for Wiskott - Aldrich Syndrome
13. Gene therapy for monogenic disorders of the bone marrow
14. Combined immunodeficiency due to JAK3 mutation in a child presenting with skin granuloma
15. A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
16. Successful RAG1-SCID gene therapy depends on the level of RAG1 expression
17. Multicenter experience in hematopoietic stem cell transplantation for serious complications of common variable immunodeficiency
18. Hematopoietic SCT in Europe: data and trends in 2011
19. KEYNOTE 3: GENE THERAPY FOR PRIMARY IMMUNODEFICIENCIES: Sponsored by Aspen Australia
20. Tandem mass spectrometry, but not T-cell receptor excision circle analysis, identifies newborns with late-onset adenosine deaminase deficiency
21. Induced pluripotent stem cells and primary immunodeficiencies: A new frontier reached, a new world beyond?
22. Bipotent Lymphoid Progenitors Independently Maintain Long-Term Genetically Engineered T and NK Cell Production in Humans
23. Immunodeficiency and disseminated mycobacterial infection associated with homozygous nonsense mutation of IKKβ
24. Signal transducer and activator of transcription 5 tyrosine phosphorylation for the diagnosis and monitoring of patients with severe combined immunodeficiency
25. Sequence Analysis of TNFRSF13b, Encoding TACI, in Patients with Systemic Lupus Erythematosus
26. Gene therapy in primary immunodeficiencies
27. The C76R transmembrane activator and calcium modulator cyclophilin ligand interactor mutation disrupts antibody production and B-cell homeostasis in heterozygous and homozygous mice
28. Transplantation of hematopoietic stem cells and long-term survival for primary immunodeficiencies in Europe: Entering a new century, do we do better?
29. Improving cellular therapy for primary immune deficiency diseases: Recognition, diagnosis, and management
30. The European Society for Immunodeficiencies (ESID) Registry Working Definitions for the Clinical Diagnosis of Inborn Errors of Immunity
31. Loss-of-function nuclear factor κB subunit 1 (NFKB1) variants are the most common monogenic cause of common variable immunodeficiency in Europeans
32. Umbilical cord blood transplantation without in vivo T-cell depletion for children with MHC class II deficiency
33. Non-Clinical Efficacy and Safety Studies on G1XCGD, a Lentiviral Vector for Ex Vivo Gene Therapy of X-Linked Chronic Granulomatous Disease
34. Pre-clinical safety and efficacy evaluation of GMP lentiviral vector in preparation for a clinical trial of hematopoietic stem cell gene therapy in MPS IIIA
35. HEMATOPOIETIC STEM CELL GENE THERAPY AS A NOVEL THERAPEUTIC APPROACH FOR SEVERE CROHN'S DISEASE ASSOCIATED WITH NOD2-DEFICIENCY.
36. New Molecular Surrogate Assay for Genotoxicity Assessment of Gene Therapy Vectors (SAGA)
37. 690. Development of a Clinical Lentiviral Vector for Gene Therapy of SCID-X1
38. How to use immunoglobulin levels in investigating immune deficiencies
39. Stem cell transplantation in severe congenital neutropenia: an analysis from the European Society for Blood and Marrow Transplantation
40. G-CSF Vs Haematopoietic Stem Cell Transplantation in Severe Congenital Neutropenia with ELANE Mutation: Role of G-CSF Dose. a Retrospective Controlled Study on Behalf of Saawp (Severe Aplastic Anemia Working Party) of the EBMT, of the Stem Cell Transplant for Immunodeficiency Group in Europe (SCETIDE), of the Severe Chronic Neutropenia French Registry (SCNFR) and Italian Neutropenia Registry (INR)
41. Enforced expression of RPS19 derived from human elongation factor 1A short (EFS) promoter corrects the anemia and lethal bone marrow failure in mouse model for RPS19-deficient diamond-blackfan anemia
42. 243. Pre-Clinical Development of Lentiviral Gene Therapy for X-Linked Severe Combined Immunodeficiency
43. 278. Successful Correction of Rag1 Severe Combined Immunedefciency at Clinically Relevant Vector Copy Numbers
44. 237. Genetic Therapy for Perforin Deficiency Associated Hemophagocytic Lymphohistiocytosis Requires High Level Expression of the Perforin Gene for Adequate Correction
45. Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency
46. Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
47. HSCT for DOCK8 Deficiency - an International Study on 74 Patients
48. Pegademase bovine (PEG-ADA) for the treatment of infants and children with severe combined immunodeficiency (SCID
49. Development of gene therapy: potential in severe combined immunodeficiency due to adenosine deaminase deficiency
50. Outcomes Of Haematopoietic Stem Cell Transplantation (HSCT) for Severe Congenital Neutropenia (SCN): Preliminary Results
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