Short Stature in Partially Corrected X-linked Severe Combined Immunodeficiency - Suboptimal Response to Growth Hormone
نویسندگان
چکیده
منابع مشابه
Short stature in Noonan syndrome: response to growth hormone therapy.
BACKGROUND Growth hormone (GH) has been used to promote growth in both the short and long term in a number of dysmorphic syndromes, including Turner syndrome. As this condition shares many clinical features with Noonan syndrome, it would seem logical to treat the latter group with GH. AIMS To assess the short and long term response to GH therapy in patients with Noonan syndrome. METHODS Ana...
متن کاملSevere short stature and Wolf-Hirschhorn syndrome: response to growth hormone in two cases without growth hormone deficiency
Wolf-Hirschhorn syndrome (WHS) is a rare congenital disorder occurring in approximately 1/50 000 births, with marked pre- and postnatal growth failure. WHS results from the hemizygous deletion encompassing the 4p16.3 region. This report of two children with WHS shows that growth hormone treatment in selected children with WHS and severe short stature may have a substantial effect on long-term g...
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Abbreviations: SCID-X1: X-linked Severe Combined Immunodeficiency; γc: Cytokine Receptor Common Gamma Chain; HSC: Hematopoietic Stem Cells; ab: Antibody; wt: Wild-type; NK cell: Natural Killer Cell; HLA: Human Leukocyte Antigen; SIN: Selfinactivating; HSV-TK: Thymidine kinase of Herpes Simplex Virus; ER: Endoplasmatic Reticulum; MFI: Mean Fluorescence Intensity; CDC: Complement-dependent Cytoto...
متن کاملEfficacy of gene therapy for X-linked severe combined immunodeficiency.
BACKGROUND The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain. METHODS The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-me...
متن کاملBone marrow transplantation for canine X-linked severe combined immunodeficiency.
Canine X-linked severe combined immunodeficiency (XSCID) is due to mutations in the common gamma chain which is a subunit of the receptors of IL-2, IL-4, IL-7, IL-9 and IL-15. Bone marrow transplantation (BMT) of human XSCID patients without pretransplant conditioning (cytoablation) results in engraftment of donor T-cells and reconstitution of T-cell function but engraftment of few, if any, don...
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ژورنال
عنوان ژورنال: Journal of Pediatric Endocrinology and Metabolism
سال: 2008
ISSN: 2191-0251,0334-018X
DOI: 10.1515/jpem.2008.21.11.1057