952 IMMUNE RECONSTITUTION IN ADENOSINE DEAMINASE DEFICIENT SEVERE COMBINED IMMUNE DEFICIENCY
نویسندگان
چکیده
منابع مشابه
Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase-deficient severe combined immune deficiency.
Genetic deficiency of adenosine deaminase (ADA) can cause profound lymphopenia and result in the clinical presentation of severe combined immune deficiency (SCID). However, because of the ubiquitous expression of ADA, ADA-deficient patients often present also with nonimmunologic clinical problems, affecting the skeletal, central nervous, endocrine, and gastrointestinal systems. We now report th...
متن کاملT lymphocyte ontogeny in adenosine deaminase-deficient severe combined immune deficiency after treatment with polyethylene glycol-modified adenosine deaminase.
Adenosine deaminase (ADA) deficiency causes severe combined immune deficiency (SCID) by interfering with the metabolism of deoxyadenosine, which is toxic to T lymphocytes at all stages of differentiation. Enzyme replacement with polyethylene glycol-modified ADA (PEG-ADA) has been previously shown to correct deoxyadenosine metabolism and improve mitogen-induced T lymphocyte proliferation. We stu...
متن کاملT Lymphocyte Ontogeny in Adenosine Deaminase - deficient Severe Combined Immune Deficiency after Treatment with Polyethylene Glycol - modified Adenosine
Adenosine deaminase (ADA) deficiency causes severe combined immune deficiency (SCID) by interfering with the metabolism of deoxyadenosine, which is toxic to T lymphocytes at all stages of differentiation. Enzyme replacement with polyethylene glycol-modified ADA (PEG-ADA) has been previously shown to correct deoxyadenosine metabolism and improve mitogen-induced T lymphocyte proliferation. We stu...
متن کاملCLINICAL TRIALS AND OBSERVATIONS Myeloid dysplasia and bone marrow hypocellularity in adenosine deaminase- deficient severe combined immune deficiency
1Disorders of Immunity Section, Genetics and Molecular Biology Branch, National Human Genome Research Institute, Bethesda, MD; 2Department of Laboratory Medicine, Clinical Center, National Institutes of Health, Bethesda, MD; 3Pediatric Oncology Branch, Center for Cancer Research, National Cancer Institute, Bethesda, MD; 4Allergy and Immunology Section, Department of Pediatrics, Baylor College o...
متن کاملGene therapy for adenosine deaminase-deficient severe combined immune deficiency: clinical comparison of retroviral vectors and treatment plans.
We conducted a gene therapy trial in 10 patients with adenosine deaminase (ADA)-deficient severe combined immunodeficiency using 2 slightly different retroviral vectors for the transduction of patients' bone marrow CD34(+) cells. Four subjects were treated without pretransplantation cytoreduction and remained on ADA enzyme-replacement therapy (ERT) throughout the procedure. Only transient (mont...
متن کاملذخیره در منابع من
با ذخیره ی این منبع در منابع من، دسترسی به آن را برای استفاده های بعدی آسان تر کنید
ژورنال
عنوان ژورنال: Pediatric Research
سال: 1981
ISSN: 0031-3998,1530-0447
DOI: 10.1203/00006450-198104001-00977