نتایج جستجو برای: diamond syndrome

تعداد نتایج: 641805  

Journal: :Cell stem cell 2016
Noemi A Zambetti Zhen Ping Si Chen Keane J G Kenswil Maria A Mylona Mathijs A Sanders Remco M Hoogenboezem Eric M J Bindels Maria N Adisty Paulina M H Van Strien Cindy S van der Leije Theresia M Westers Eline M P Cremers Chiara Milanese Pier G Mastroberardino Johannes P T M van Leeuwen Bram C J van der Eerden Ivo P Touw Taco W Kuijpers Roland Kanaar Arjan A van de Loosdrecht Thomas Vogl Marc H G P Raaijmakers

Mesenchymal niche cells may drive tissue failure and malignant transformation in the hematopoietic system, but the underlying molecular mechanisms and relevance to human disease remain poorly defined. Here, we show that perturbation of mesenchymal cells in a mouse model of the pre-leukemic disorder Shwachman-Diamond syndrome (SDS) induces mitochondrial dysfunction, oxidative stress, and activat...

Journal: :Hematology. American Society of Hematology. Education Program 2005
Blanche P Alter

Aplastic anemia may be inherited or acquired. The distinction between these lies not in the age of the patient, but in the clinical and laboratory diagnoses. Adult hematologists must consider adult presentations of the inherited disorders, in order to avoid incorrect management of their patients. Physicians for adult patients must also realize that children with inherited disorders now survive ...

Journal: :Blood 1988
S Ozsoylu

I read with interest the recent article by Lenarsky et al’ about bone marrow transplantation (BMT) for Diamond-Blackfan syndrome. I reported a case of constitutional pure red cell aplasia (CPRCA) refractory to conventional prednisone (2 mg/kg) administration treated with high-dose intravenous (IV) methylprednisolone (HIVMP) (30 mg/kg for 3 days, 20 mg/kg for 4 days, then subsequently 10, 5, and...

Journal: :The Journal of pediatrics 2005
Joshua D Groman Barbara Karczeski Molly Sheridan Terry E Robinson M Daniele Fallin Garry R Cutting

OBJECTIVE To determine which features of incomplete or "nonclassic" forms of cystic fibrosis (CF) are associated with deleterious CF transmembrane conductance regulator gene ( CFTR ) mutations, and to explore other etiologies for features not associated with deleterious CFTR mutations. STUDY DESIGN Clinical features were compared between 57 patients with deleterious mutations in each CFTR and...

Journal: :Genes & development 2011
Andrew J Finch Christine Hilcenko Nicolas Basse Lesley F Drynan Beatriz Goyenechea Tobias F Menne Africa González Fernández Paul Simpson Clive S D'Santos Mark J Arends Jean Donadieu Christine Bellanné-Chantelot Michael Costanzo Charles Boone Andrew N McKenzie Stefan M V Freund Alan J Warren

Removal of the assembly factor eukaryotic initiation factor 6 (eIF6) is critical for late cytoplasmic maturation of 60S ribosomal subunits. In mammalian cells, the current model posits that eIF6 release is triggered following phosphorylation of Ser 235 by activated protein kinase C. In contrast, genetic studies in yeast indicate a requirement for the ortholog of the SBDS (Shwachman-Bodian-Diamo...

2010
Babu A. Manjasetty Sunil Kumar Andrew P. Turnbull Niraj Kanti Tripathy

The functional correlation of missense mutations which cause disease remains a challenge to understanding the basis of genetic diseases. This is particularly true for proteins related to diseases for which there are no available three dimensional structures. One such disease is Shwachman Diamond syndrome SDS OMIM 260400, a multi system disease arising from loss of functional mutations. The Homo...

2008
Jeong Hee Lee Sun Hwan Bae Jeong Jin Yu Ran Lee Yeo Min Yun Eun Young Song

Shwachman-Diamond syndrome (SDS) is an autosomal recessive genetic disorder, consisting of exocrine pancreatic insufficiency, chronic neutropenia, neutrophil chemotaxis defects, metaphyseal dysostosis, short stature, dental caries, and multiple organ involvements. Although SDS is the second most common hereditary abnormality of exocrine pancreas following cystic fibrosis in the Western countrie...

2012
Niraj Shenoy Rachel Kessel Tushar D Bhagat Sanchari Bhattacharyya Yiting Yu Christine Mcmahon Amit Verma

Ribosomes are essential components of the protein translation machinery and are composed of more than 80 unique large and small ribosomal proteins. Recent studies show that in addition to their roles in protein translation, ribosomal proteins are also involved in extra-ribosomal functions of DNA repair, apoptosis and cellular homeostasis. Consequently, alterations in the synthesis or functionin...

Journal: :Journal of pediatric gastroenterology and nutrition 2005
Rosa M Lima Elísio Costa Cristina Rocha Emília Vieira Rosário dos Santos José Barbot Herculano Rocha

Shwachman-Diamond syndrome (SDS) a rare autosomal recessive disorder described first time 1964 (1), is characterized by the association of exocrine pancreatic and bone marrow dysfunction. Other systemic findings (skeletal, liver and psychomotor) or problems secondary to bone marrow dysfunction may also be detected (1–4). Intermittent or persistent neutropenia is the most common hematologic find...

2010
Shahinaz M. Gadalla Richard Cawthon Neelam Giri Blanche P. Alter Sharon A. Savage

Telomeres, the nucleotide repeats and protein complex at chromosome ends, are required for chromosomal stability and are important markers of aging. Patients with dyskeratosis congenita (DC), an inherited bone marrow failure syndrome (IBMFS), have mutations in telomere biology genes, and very short telomeres. There are limited data on intra-individual telomere length (TL) variability in DC and ...

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