نتایج جستجو برای: glucocerebrosidase

تعداد نتایج: 825  

2015
Juan Marcos Mucci Paula Rozenfeld

Gaucher, the most prevalent lysosomal disorder, is an autosomal recessive inherited disorder due to a deficiency of glucocerebrosidase. Glucocerebrosidase deficiency leads to the accumulation of glucosylceramide primarily in cells of mononuclear-macrophage lineage. Clinical alterations are visceral, hematological, and skeletal. Bone disorder in Gaucher disease produces defects on bone metabolis...

2007
Naoki Tanaka Hiroshi Saito Toshiro Ito Kayoko Momose Fumihiro Ishida Hiroyuki Ida

A 27-year-old womanwas admitted for further examination of thrombocytopenia. Symptomswere absent, but physical examination demonstrated hepatosplenomegaly without neurological abnormalities. Bone marrow examination revealed many Gaucher cells, and glucocerebrosidase activity from cultured skin fibroblasts was markedly reduced. A 1448C (L444P) mutation was detected on one allele of the glucocere...

2018
Mia Horowitz

Recently, the development of Parkinson’s disease (PD) has Background: been linked to a number of genetic risk factors, of which the most common is glucocerebrosidase (GBA) mutations. We investigated PD and Gaucher Disease (GD) patient derived skin Methods: fibroblasts using biochemistry assays. PD patient derived skin fibroblasts have normal glucocerebrosidase Results: (GCase) activity, whilst ...

2016
Gertjan Kramer Wouter Wegdam Wilma Donker‐Koopman Roelof Ottenhoff Paulo Gaspar Marri Verhoek Jessica Nelson Tanit Gabriel Wouter Kallemeijn Rolf G. Boot Jon D. Laman Johannes P.C. Vissers Timothy Cox Elena Pavlova Mary Teresa Moran Johannes M. Aerts Marco van Eijk

Gaucher disease is caused by inherited deficiency of lysosomal glucocerebrosidase. Proteome analysis of laser-dissected splenic Gaucher cells revealed increased amounts of glycoprotein nonmetastatic melanoma protein B (gpNMB). Plasma gpNMB was also elevated, correlating with chitotriosidase and CCL18, which are established markers for human Gaucher cells. In Gaucher mice, gpNMB is also produced...

2017

Recently, the development of Parkinson’s disease (PD) has Background: been linked to a number of genetic risk factors, of which the most common is glucocerebrosidase (GBA) mutations. We investigated PD and Gaucher Disease (GD) patient derived skin Methods: fibroblasts using biochemistry assays. PD patient derived skin fibroblasts have normal glucocerebrosidase Results: (GCase) activity, whilst ...

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...

2017
Lucy M Collins Janelle Drouin-Ouellet Wei-Li Kuan Timothy Cox Roger A Barker Dario Besusso Mia Horowitz Lucy Collins

Background: Recently, the development of Parkinson's disease (PD) has been linked to a number of genetic risk factors, of which the most common is glucocerebrosidase (GBA) mutations. Methods: We investigated PD and Gaucher Disease (GD) patient derived skin fibroblasts using biochemistry assays. Results: PD patient derived skin fibroblasts have normal glucocerebrosidase (GCase) activity, whilst ...

Journal: :Parkinsonism & Related Disorders 2014

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