نتایج جستجو برای: gaucher disease

تعداد نتایج: 1490299  

2016
Wendy Westbroek Matthew Nguyen Marina Siebert Taylor Lindstrom Robert A Burnett Elma Aflaki Olive Jung Rafael Tamargo Jorge L Rodriguez-Gil Walter Acosta An Hendrix Bahafta Behre Nahid Tayebi Hideji Fujiwara Rohini Sidhu Benoit Renvoise Edward I Ginns Amalia Dutra Evgenia Pak Carole Cramer Daniel S Ory William J Pavan Ellen Sidransky

Glucocerebrosidase is a lysosomal hydrolase involved in the breakdown of glucosylceramide. Gaucher disease, a recessive lysosomal storage disorder, is caused by mutations in the gene GBA1 Dysfunctional glucocerebrosidase leads to accumulation of glucosylceramide and glycosylsphingosine in various cell types and organs. Mutations in GBA1 are also a common genetic risk factor for Parkinson diseas...

2017
Mario de la Mata David Cotán Manuel Oropesa-Ávila Marina Villanueva-Paz Isabel de Lavera Mónica Álvarez-Córdoba Raquel Luzón-Hidalgo Juan M. Suárez-Rivero Gustavo Tiscornia José A. Sánchez-Alcázar

BACKGROUND Gaucher disease (GD) is caused by mutations in the GBA1 gene which encodes lysosomal β-glucocerebrosidase (GCase). In GD, partial or complete loss of GCase activity causes the accumulation of the glycolipids glucosylceramide (GlcCer) and glucosylsphingosine in the lysosomes of macrophages. In this manuscript, we investigated the effects of glycolipids accumulation on lysosomal and mi...

Journal: :Journal of Clinical Movement Disorders 2017

2016
Christine Serratrice Sebastian Carballo Jacques Serratrice Jérome Stirnemann

INTRODUCTION Gaucher disease is the first lysosomal disease to benefit from enzyme replacement therapy, thus serving as model for numerous other lysosomal diseases. Alglucerase was the first glucocerebrosidase purified from placental extracts, and this was then replaced by imiglucerase - a Chinese hamster ovary cell-derived glucocerebrosidase. AIM The aim was to review the evidence underlying...

Journal: :Lijecnicki vjesnik 2014
Marijan Merkler Iveta Simić Ivan Pećin Diana Muacević-Katanec Nediljko Sucur Zeljko Reiner

Gaucher disease is an autosomal recessive disorder, characterized by decreased levels of the lysosomal enzyme glucocerebrosidase. This deficiency results in a decreased breakdown of this glycosphingolipid glucocerebroside, which accumulates in the lysosomes of the monocyte-macrophage system. It is the most common form of sphingolipidosis. Clinically, the principle signs of Gaucher's disease are...

Journal: :BMC Oral Health 2003
Stuart L Fischman Deborah Elstein Harold Sgan-Cohen Jonathan Mann Ari Zimran

BACKGROUND: This study was conducted to determine whether patients with Gaucher disease had significant dental pathology because of abnormal bone structure, pancytopenia, and coagulation abnormalities. METHODS: Each patient received a complete oral and periodontal examination in addition to a routine hematological evaluation. RESULTS: Gaucher patients had significantly fewer carious lesions tha...

2017
Elma Aflaki Daniel K. Borger Richard J. Grey Martha Kirby Stacie Anderson Grisel Lopez Ellen Sidransky

Gaucher disease, the inherited deficiency of lysosomal glucocerebrosidase, is characterized by the presence of glucosylceramide-laden macrophages resulting from impaired digestion of aged erythrocytes or apoptotic leukocytes. Studies of macrophages from patients with type 1 Gaucher disease with genotypes N370S/N370S, N370S/L444P or N370S/c.84dupG revealed that Gaucher macrophages have impaired ...

Journal: :Blood 1994
Y Liel A Rudich O Nagauker-Shriker T Yermiyahu R Levy

Gaucher disease patients are occasionally affected by chronic or fulminant infections. Since Gaucher cells originate from tissue phagocytes, we studied the functional implications of glucocerbroside accumulation on phagocytes in Gaucher disease patients. Circulating monocytes and granulocytes from nine type I Gaucher disease patients, and matched controls, were studied. Evaluation of phagocytic...

Journal: :International journal of medical science and clinical research studies 2022

Splenectomy can treat conditions characterized by hemolysis or thrombocytopenia caused autoantibodies splenic reticuloendothelial function, as well disorders massive splenomegaly and hypersplenism with cytopenias, such hereditary spherocytosis, transfusion-dependent thalassemia, immune thrombocytopenia, autoimmune hemolytic anemia, marginal zone lymph is no longer used for Hodgkin disease stagi...

2016
Gaetano Giuffrida Rita Lombardo Ernesto Di Francesco Laura Parrinello Francesco Di Raimondo Agata Fiumara

BACKGROUND Gaucher disease is one of the most common lipid-storage disorders, affecting approximately 1 in 75,000 births. Enzyme replacement therapy with recombinant glucocerebrosidase is currently considered the first-line treatment choice for patients with symptomatic Gaucher disease type 1. Oral substrate reduction therapy is generally considered a second-line treatment option for adult pati...

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