نتایج جستجو برای: diamond syndrome

تعداد نتایج: 641805  

Journal: :Haematologica 2015
Noemi A Zambetti Eric M J Bindels Paulina M H Van Strien Marijke G Valkhof Maria N Adisty Remco M Hoogenboezem Mathijs A Sanders Johanna M Rommens Ivo P Touw Marc H G P Raaijmakers

Shwachman-Diamond syndrome is a congenital bone marrow failure disorder characterized by debilitating neutropenia. The disease is associated with loss-of-function mutations in the SBDS gene, implicated in ribosome biogenesis, but the cellular and molecular events driving cell specific phenotypes in ribosomopathies remain poorly defined. Here, we established what is to our knowledge the first ma...

Journal: :The Journal of clinical investigation 2017
Raphael Carapito Martina Konantz Catherine Paillard Zhichao Miao Angélique Pichot Magalie S Leduc Yaping Yang Katie L Bergstrom Donald H Mahoney Deborah L Shardy Ghada Alsaleh Lydie Naegely Aline Kolmer Nicodème Paul Antoine Hanauer Véronique Rolli Joëlle S Müller Elisa Alghisi Loïc Sauteur Cécile Macquin Aurore Morlon Consuelo Sebastia Sancho Patrizia Amati-Bonneau Vincent Procaccio Anne-Laure Mosca-Boidron Nathalie Marle Naël Osmani Olivier Lefebvre Jacky G Goetz Sule Unal Nurten A Akarsu Mirjana Radosavljevic Marie-Pierre Chenard Fanny Rialland Audrey Grain Marie-Christine Béné Marion Eveillard Marie Vincent Julien Guy Laurence Faivre Christel Thauvin-Robinet Julien Thevenon Kasiani Myers Mark D Fleming Akiko Shimamura Elodie Bottollier-Lemallaz Eric Westhof Claudia Lengerke Bertrand Isidor Seiamak Bahram

Shwachman-Diamond syndrome (SDS) (OMIM #260400) is a rare inherited bone marrow failure syndrome (IBMFS) that is primarily characterized by neutropenia and exocrine pancreatic insufficiency. Seventy-five to ninety percent of patients have compound heterozygous loss-of-function mutations in the Shwachman-Bodian-Diamond syndrome (sbds) gene. Using trio whole-exome sequencing (WES) in an sbds-nega...

2017
Ling Zhang

Bone marrow failure syndromes (BMFS) are a cluster of inherited or acquired disorders characterized by peripheral cytopenia due to a decrease in hematopoietic progenitors or dysregulated hematopoiesis. Inherited bone marrow failure syndromes are mainly found in pediatric group, encompassing Diamond Blackfan anemia (DBA), Fanconi anemia (FA), congenital sideroblastic anemia (CSA), congenital neu...

2007
FRANCESCO MOLINARI

Shwachman Diamond Syndrome is a rare congenital disorder characterized by pancreatic insufficiency, bone marrow dysfunction and skeletal abnormalities. No specific test is available for a definitive diagnosis for SDS. In the presence of clinical features of SDS, pancreatic insufficiency should be tested for or lipomatosis radiologically demonstrated. New directions might be found in genetic ana...

Journal: :Folia biologica 2016
A Kubik-Zahorodna B Schuster I Kanchev R Sedláček

Diamond-Blackfan anaemia is a rare disease caused by insufficient expression of ribosomal proteins and is characterized by erythroid hypoplasia often accompanied by growth retardation, congenital craniofacial and limb abnormalities. In addition, Diamond-Blackfan anaemia patients also exhibit a number of behavioural abnormalities. In this study we describe the behavioural effects observed in a n...

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