Frederick D. Goldman
A5020617631- Hematopoietic Stem Cell Transplantation
- Immunodeficiency and Autoimmune Disorders
- Telomeres, Telomerase, and Senescence
- Immune Cell Function and Interaction
- Virus-based gene therapy research
- Cytomegalovirus and herpesvirus research
- Skin Protection and Aging
- Acute Lymphoblastic Leukemia research
- Blood disorders and treatments
- CRISPR and Genetic Engineering
- CAR-T cell therapy research
- Childhood Cancer Survivors' Quality of Life
- Pneumocystis jirovecii pneumonia detection and treatment
- Acute Myeloid Leukemia Research
- Mesenchymal stem cell research
- Neutropenia and Cancer Infections
- DNA Repair Mechanisms
- T-cell and B-cell Immunology
- Cystic Fibrosis Research Advances
- Neutrophil, Myeloperoxidase and Oxidative Mechanisms
- Renal Transplantation Outcomes and Treatments
- Hemoglobinopathies and Related Disorders
- Prenatal Screening and Diagnostics
- Platelet Disorders and Treatments
- Autoimmune and Inflammatory Disorders Research
University of Alabama at Birmingham
2016-2025
Children's of Alabama
2011-2024
RELX Group (United States)
2021
University of Alabama
2009-2013
Penn State Milton S. Hershey Medical Center
2011
University of Iowa
1994-2010
University of Iowa Stead Family Children’s Hospital
2006-2009
Magee-Womens Hospital
2009
University of Iowa Hospitals and Clinics
1996-2006
Children's Oncology Group
2006
The Primary Immune Deficiency Treatment Consortium was formed to analyze the results of hematopoietic-cell transplantation in children with severe combined immunodeficiency (SCID) and other primary immunodeficiencies. Factors associated a good outcome need be identified order design safer more effective curative therapy, particularly for SCID diagnosed at birth.
<h3>Background</h3> Immunodysregulation polyendocrinopathy enteropathy x-linked (IPEX) syndrome is a monogenic autoimmune disease caused by <i>FOXP3</i> mutations. Because it rare disease, the natural history and response to treatments, including allogeneic hematopoietic stem cell transplantation (HSCT) immunosuppression (IS), have not been thoroughly examined. <h3>Objective</h3> This analysis sought evaluate onset, progression, long-term outcome of 2 main treatments in IPEX survivors....
We describe outcomes after allogeneic transplantation in 34 patients with dyskeratosis congenita who underwent between 1981 and 2009. The median age at was 13 years (range, 2 to 35). Approximately 50% of transplantations were from related donors. Bone marrow the predominant source stem cells (24 34). day-28 probability neutrophil recovery 73% day-100 platelet 72%. grade II IV acute GVHD 3-year chronic graft-versus-host disease 24% 37%, respectively. 10-year survival 30%; 14 alive last...
Mutations of the Janus family kinase JAK3 gene cause severe combined immunodeficiency (SCID). deficiency in humans is characterized by absence circulating T cells and natural killer (NK) with normal numbers poorly functioning B (T(-)B(+)NK(-)). Using SCID patient-specific induced pluripotent stem (iPSCs) a cell vitro differentiation system, we demonstrate complete block early development JAK3-deficient cells. Correction mutation CRISPR/Cas9-enhanced targeting restores development, including...
Congenital pancytopenia is a rare and often lethal condition. Current knowledge of lymphoid hematopoietic development in mice, as well understanding regulators human hematopoiesis, have led to the recent discovery genetic causes bone marrow failure disorders. However, absence mutations specific genes or distinct clinical phenotype, many cases aplastic anemia are labeled idiopathic, while congenital immune deficiencies described combined deficiency.We describe case 33-week gestation age male...
Primary Immune Regulatory Disorders (PIRD) are an expanding group of diseases caused by gene defects in several different immune pathways, such as regulatory T cell function. Patients with PIRD develop clinical manifestations associated diminished and exaggerated responses. Management these patients is complicated; oftentimes immunosuppressive therapies insufficient, may require hematopoietic transplant (HCT) for treatment. Analysis HCT data have previously focused on a single defect. This...
The past 4 decades have seen substantial changes in allogeneic blood or marrow transplantation (BMT) practice, with the overarching goal of expanding eligible patient pool while optimizing disease-free survival.To determine trends life expectancy and cause-specific late mortality after BMT performed over a 40-year period.A retrospective cohort study 4741 individuals who lived 2 more years between January 1, 1974, December 31, 2014, was conducted at City Hope, University Minnesota, Alabama...
Severe combined immune deficiency (SCID) is a heterogeneous disorder characterized by profound defects in cellular and humoral immunity. We report here an infant with clinical laboratory features of SCID selective CD4 lymphopenia lack CD28 expression on CD8(+) T cells. cells from this patient showed poor blastogenic responses to various mitogens IL-2. Other cell antigen receptor- induced responses, including upregulation CD69, were similarly inhibited. However, more proximal receptor...
Surface expression of the CD45 tyrosine phosphatase is essential for T cell antigen receptor (TCR) to couple optimally with its second messenger pathways. may be required dephosphorylate a TCR-activated protein kinase, which then transduces an activation signal from TCR. A chimeric molecule that contained extracellular and transmembrane sequences allele major histocompatibility class I cytoplasmic restored TCR signaling in CD45-deficient mutant line. Thus, complex domain not machinery.
C h ronic graft-versus-host disease (GVHD) is a major complication of allogeneic bone marrow transplantation.Both the and medications used to treat it are associated with significant morbidity mort l i t y.The manifestations chronic GVHD often resemble those autoimmune disorders.Hydro x y c o roquine (HCQ) 4-aminoquinoline antimalarial drug for treatment diseases.HCQ interf e res antigen processing presentation, cytokine production, cytotoxicity synergistic cyclosporine rolimus in...