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The New England Journal of Medicine, ISSN 0028-4793, 07/2014, Volume 371, Issue 5, pp. 434 - 446
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2016, Volume 137, Issue 2, pp. AB23 - AB23
  Here we report an infant girl who presented with initial absent TRECs and mild T-cell lymphopenia after in utero exposure to 7 cycles of 5-fluorouracil,... 
Allergy and Immunology | Chemotherapy | Cancer | Flow cytometry | T cell receptors
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2010, Volume 126, Issue 5, pp. 1000 - 1005
Background Immune dysregulation, polyendocrinopathy, enteropathy, X-linked (IPEX) syndrome is characterized by severe systemic autoimmunity caused by mutations... 
Allergy and Immunology | regulatory T cells | Hematopoietic cell transplantation | autoimmunity | immune reconstitution | immunodeficiency | nonmyeloablative conditioning regimen | immune dysregulation | Autoimmunity | Immune dysregulation | Immunodeficiency | Immune reconstitution | Nonmyeloablative conditioning regimen | Regulatory T cells | FOXP3 EXPRESSION | DIARRHEA | BONE-MARROW-TRANSPLANTATION | IPEX SYNDROME | IMMUNOLOGY | VERSUS-HOST-DISEASE | ALLERGY | T-CELLS | Graft vs Host Disease - therapy | Myeloablative Agonists - therapeutic use | Whole-Body Irradiation | Humans | Immunosuppressive Agents - therapeutic use | Infant | Male | Flow Cytometry | Hematopoietic Stem Cell Transplantation - adverse effects | Genetic Diseases, X-Linked - genetics | Transplantation Conditioning - methods | Child | Graft vs Host Disease - etiology | Genetic Diseases, X-Linked - physiopathology | Immunologic Deficiency Syndromes - therapy | Cell Separation | Vidarabine - analogs & derivatives | Vidarabine - therapeutic use | Forkhead Transcription Factors - genetics | Reverse Transcriptase Polymerase Chain Reaction | Syndrome | Graft vs Host Disease - pathology | Cyclosporine - therapeutic use | Mycophenolic Acid - analogs & derivatives | Mycophenolic Acid - therapeutic use | Immunologic Deficiency Syndromes - physiopathology | Adolescent | Immunologic Deficiency Syndromes - genetics | Hematopoietic Stem Cell Transplantation - methods | Genetic Diseases, X-Linked - therapy | Medical colleges | Granulocyte colony-stimulating factor | Oncology, Experimental | Transplantation | Research | Macrophage colony stimulating factor | Hematopoietic stem cells | Cancer | Bone marrow | Mutation | Transplants & implants | Mortality | Stem cells | Index Medicus | Abridged Index Medicus | Regulatory T-cells
Journal Article
Journal of Clinical Investigation, ISSN 0021-9738, 05/2017, Volume 127, Issue 5, pp. 1689 - 1699
BACKGROUND. Autologous hematopoietic stem cell transplantation (HSCT) of gene-modified cells is an alternative to enzyme replacement therapy (ERT) and... 
MEDICINE, RESEARCH & EXPERIMENTAL | THERAPY | SCID PATIENTS | RETROVIRAL VECTORS | RECONSTITUTION | EXPRESSION | ADA | TRANSPLANTATION | Autografts | Genetic Therapy | Severe Combined Immunodeficiency - enzymology | Transduction, Genetic | Agammaglobulinemia - genetics | Adenosine Deaminase - genetics | Agammaglobulinemia - therapy | Humans | Adenosine Deaminase - biosynthesis | Child, Preschool | Hematopoietic Stem Cell Transplantation | Retroviridae | Severe Combined Immunodeficiency - therapy | Infant | Male | Gene Expression Regulation, Enzymologic | Severe Combined Immunodeficiency - genetics | Agammaglobulinemia - enzymology | Adolescent | Female | Adenosine Deaminase - deficiency | Genetic Vectors | Child | Treatment outcome | Care and treatment | Usage | Safety and security measures | Analysis | Adenosine deaminase deficiency | Genetic aspects | Genetic engineering | Transplantation | Research | Hematopoietic stem cells | Pediatrics | Intravenous administration | Severe combined immunodeficiency | Transplants & implants | ADA gene | Copy number | Stem cell transplantation | Clinical trials | Genomes | Immunology | Lymphocytes | Peripheral blood mononuclear cells | Genetics | Busulfan | Research centers | Age | CD34 antigen | Enzymes | Adenosine | Enrollments | Research & development--R&D | Immunodeficiency | Immune reconstitution | Data processing | Adenosine deaminase | Gene expression | Medical screening | Hemopoiesis | Children & youth | Medicine | Studies | Hospitals | Stem cells | Product development | Gene therapy | Leukocytes (granulocytic) | Mitogens
Journal Article
Journal of Allergy and Clinical Immunology, The, ISSN 0091-6749, 2013, Volume 132, Issue 1, pp. 223 - 226
Journal Article
Molecular Genetics and Metabolism, ISSN 1096-7192, 02/2016, Volume 117, Issue 2, pp. S18 - S18
Journal Article
The Journal of Allergy and Clinical Immunology: In Practice, ISSN 2213-2198, 09/2018, Volume 6, Issue 5, pp. 1749 - 1751
Immune modulation has been used in lysosomal storage disorders, most notably Pompe disease.1 The Duke University immune tolerance induction protocol was shown... 
TOLERANCE INDUCTION | ALLERGY | IMMUNOLOGY | Urine | Enzymes | Immunoglobulins | Immune response | Disease | Immunomodulation | Exons | Clinical trials | Lysosomal storage diseases | Gene deletion | Patients | Immunological tolerance | Genetic screening | Clonal deletion | Analgesics | Mutation
Journal Article
Journal of Clinical Immunology, ISSN 0271-9142, 10/2019, Volume 39, Issue 7, pp. 653 - 667
Journal Article
Journal Article
Blood, ISSN 0006-4971, 10/2018, Volume 132, Issue 17, pp. 1737 - 1749
The Primary Immune Deficiency Treatment Consortium (PIDTC) performed a retrospective analysis of 662 patients with severe combined immunodeficiency (SCID) who... 
CRITERIA | SEVERE COMBINED IMMUNODEFICIENCY | BONE-MARROW-TRANSPLANTATION | DISEASE | HCT | EXPERIENCE | COHORT | STEM-CELL TRANSPLANTATION | HEMATOLOGY | RECONSTITUTION | Plenary Paper | 100
Journal Article
Pediatric Blood & Cancer, ISSN 1545-5009, 11/2015, Volume 62, Issue 11, pp. 2047 - 2049
Journal Article
The Pediatric infectious disease journal, ISSN 0891-3668, 1/2012, Volume 31, Issue 1, pp. 95 - 97
This retrospective review of patients with severe combined immunodeficiency and Pneumocystis jirovecii pneumonia (PCP) evaluated the relationship between... 
pneumonia | PCP | severe combined immunodeficiency | SCID | primary immune deficiency | Pneumocystis jirovecii
Journal Article
Therapeutics and Clinical Risk Management, ISSN 1176-6336, 2010, Volume 6, Issue 1, pp. 1 - 10
Journal Article