نتایج جستجو برای: pick type c1 npc1

تعداد نتایج: 1363808  

Journal: :Molecular and Cellular Neuroscience 2014
Isabelle Buard Frank W. Pfrieger

Niemann-Pick type C disease is a rare and ultimately fatal lysosomal storage disorder with variable neurologic symptoms. The disease-causing mutations concern NPC1 or NPC2, whose dysfunction entails accumulation of cholesterol in the endosomal-lysosomal system and the selective death of specific neurons, namely cerebellar Purkinje cells. Here, we investigated whether neurodegeneration is preced...

Journal: :Traffic 2010
Cecilia Devlin Nina H Pipalia Xianghai Liao Edward H Schuchman Frederick R Maxfield Ira Tabas

Different primary lysosomal trafficking defects lead to common alterations in lipid trafficking, suggesting cooperative interactions among lysosomal lipids. However, cellular analysis of the functional consequences of this phenomenon is lacking. As a test case, we studied cells with defective Niemann-Pick C1 (NPC1) protein, a cholesterol trafficking protein whose defect gives rise to lysosomal ...

Journal: :Human molecular genetics 2003
Titta S Blom Matts D Linder Karen Snow Helena Pihko Michael W Hess Eija Jokitalo Ville Veckman Ann-Christine Syvänen Elina Ikonen

Niemann-Pick type C (NPC) disease is a fatal recessively inherited lysosomal cholesterol-sphingolipidosis. Mutations in the NPC1 gene cause approximately 95% of the cases, the rest being caused by NPC2 mutations. Here the molecular basis of a severe infantile form of the disease was dissected. The level of NPC1 protein in the patient fibroblasts was similar to that in control cells. However, th...

2015
William S. Garver Lesley de la Torre Matthew C. Brennan Li Luo David Jelinek Joseph J. Castillo David Meyre Robert A. Orlando Randall A. Heidenreich William F. Rayburn

A genome-wide association study (GWAS) and subsequent replication studies in diverse ethnic groups indicate that common Niemann-Pick C1 gene (NPC1) polymorphisms are associated with morbid-adult obesity or diabetes independent of body weight. The objectives for this prospective cross-sectional study were to determine allele frequencies for NPC1 polymorphisms (644A>G, 1926C>G, 2572A>G, and 3797G...

Journal: :Science translational medicine 2016
Thomas Kirkegaard James Gray David A Priestman Kerri-Lee Wallom Jennifer Atkins Ole Dines Olsen Alexander Klein Svetlana Drndarski Nikolaj H T Petersen Linda Ingemann David A Smith Lauren Morris Claus Bornæs Signe Humle Jørgensen Ian Williams Anders Hinsby Christoph Arenz David Begley Marja Jäättelä Frances M Platt

Lysosomal storage diseases (LSDs) often manifest with severe systemic and central nervous system (CNS) symptoms. The existing treatment options are limited and have no or only modest efficacy against neurological manifestations of disease. We demonstrate that recombinant human heat shock protein 70 (HSP70) improves the binding of several sphingolipid-degrading enzymes to their essential cofacto...

2013
Charles J. Shoemaker Kathryn L. Schornberg Sue E. Delos Corinne Scully Hassan Pajouhesh Gene G. Olinger Lisa M. Johansen Judith M. White

Ebola virus (EBOV) is an enveloped RNA virus that causes hemorrhagic fever in humans and non-human primates. Infection requires internalization from the cell surface and trafficking to a late endocytic compartment, where viral fusion occurs, providing a conduit for the viral genome to enter the cytoplasm and initiate replication. In a concurrent study, we identified clomiphene as a potent inhib...

Journal: :Journal of lipid research 2010
Mark Charman Barry E Kennedy Nolan Osborne Barbara Karten

Niemann-Pick Type C (NPC) disease is a fatal, neurodegenerative disorder, caused in most cases by mutations in the late endosomal protein NPC1. A hallmark of NPC disease is endosomal cholesterol accumulation and an impaired cholesterol homeostatic response, which might affect cholesterol transport to mitochondria and, thus, mitochondrial and cellular function. This study aimed to characterize m...

Journal: :Glia 2007
Gang Chen Hai-Min Li Yi-Ren Chen Xiao-Song Gu Shumin Duan

Niemann-Pick disease type C (NPC) is a deadly neurodegenerative disease often caused by mutation in a gene called NPC1, which results in the accumulation of unesterified cholesterol and glycosphingolipids in the endosomal-lysosomal system. Most studies on the mechanisms of neurodegeneration in NPC have focused on neurons. However, the possibility also exists that NPC1 affects neuronal functions...

نمودار تعداد نتایج جستجو در هر سال

با کلیک روی نمودار نتایج را به سال انتشار فیلتر کنید