نتایج جستجو برای: npc1

تعداد نتایج: 544  

Journal: :Blood 2014
Anneliese O Speak Danielle Te Vruchte Lianne C Davis Anthony J Morgan David A Smith Nicole M Yanjanin Louise Simmons Ralf Hartung Heiko Runz Eugen Mengel Michael Beck Jackie Imrie Elizabeth Jacklin James E Wraith Christian Hendriksz Robin Lachmann Celine Cognet Rohini Sidhu Hideji Fujiwara Daniel S Ory Antony Galione Forbes D Porter Eric Vivier Frances M Platt

Niemann-Pick type C (NPC) is a neurodegenerative lysosomal storage disorder caused by defects in the lysosomal proteins NPC1 or NPC2. NPC cells are characterized by reduced lysosomal calcium levels and impaired sphingosine transport from lysosomes. Natural killer (NK) cells kill virally infected/transformed cells via degranulation of lysosome-related organelles. Their trafficking from lymphoid ...

2012
Sarah N.R. Pressey David A. Smith Andrew M.S. Wong Frances M. Platt Jonathan D. Cooper

Niemann-Pick disease type C (NPC) is an inherited lysosomal storage disease characterised by accumulation of cholesterol and glycosphingolipids. NPC patients suffer a progressive neurodegenerative phenotype presenting with motor dysfunction, mental retardation and cognitive decline. To examine the onset and progression of neuropathological insults in NPC we have systematically examined the CNS ...

Journal: :The Biochemical journal 2005
Fannie W Chen Ronald E Gordon Yiannis A Ioannou

NPC (Niemann-Pick type C) disease is a rare lipidosis characterized by the accumulation of LDL (low-density lipoprotein)-derived non-esterified cholesterol in the E/L (endosomal/lysosomal) system. The gene products that are responsible for the two NPC complementation groups are distinct and dissimilar, yet their cellular and disease phenotypes are virtually indistinguishable. To investigate the...

2014
Dorothea Maetzel Sovan Sarkar Haoyi Wang Lina Abi-Mosleh Ping Xu Albert W. Cheng Qing Gao Maisam Mitalipova Rudolf Jaenisch

Niemann-Pick type C (NPC) disease is a fatal inherited lipid storage disorder causing severe neurodegeneration and liver dysfunction with only limited treatment options for patients. Loss of NPC1 function causes defects in cholesterol metabolism and has recently been implicated in deregulation of autophagy. Here, we report the generation of isogenic pairs of NPC patient-specific induced pluripo...

2017
Franziska Peter Michaela Trilck Michael Rabenstein Arndt Rolfs Moritz J. Frech

Data presented in this article demonstrate the generation and characterization of two novel Niemann-Pick disease Type C1 (NPC1) patient-specific induced pluripotent stem cell (iPSC) lines, related to the research article Trilck et al. (Diversity of Glycosphingolipid GM2 and Cholesterol Accumulation in NPC1 Patient-Specific iPSC-Derived Neurons; Brain Res.; 2017; 1657:52-61. doi: 10.1016/j.brain...

Journal: :International journal of molecular sciences 2018
Lynn Ebner Anne Gläser Anja Bräuer Martin Witt Andreas Wree Arndt Rolfs Marcus Frank Brigitte Vollmar Angela Kuhla

Niemann-Pick-disease type C1 (NPC1) is an autosomal-recessive cholesterol-storage disorder. Besides other symptoms, NPC1 patients develop liver dysfunction and hepatosplenomegaly. The mechanisms of hepatomegaly and alterations of lipid metabolism-related genes in NPC1 disease are still poorly understood. Here, we used an NPC1 mouse model to study an additive hepatoprotective effect of a combina...

Journal: :Journal of lipid research 2004
Patrick C Reid Naomi Sakashita Shigeki Sugii Yoshiko Ohno-Iwashita Yukiko Shimada William F Hickey Ta-Yuan Chang

Niemann-Pick type C (NPC) is a neurodegenerative disorder characterized by progressive accumulation of cholesterol, gangliosides, and other lipids in the central nervous system and visceral organs. In the NPC1 mouse model, neurodegeneration and neuronal cell loss occur before postnatal day 21. Whether neuronal cholesterol accumulation occurs in vivo before the first signs of neuronal cell loss ...

Journal: :Proceedings of the National Academy of Sciences of the United States of America 2006
S Joshua Langmade Sarah E Gale Andrey Frolov Ikuko Mohri Kinuko Suzuki Synthia H Mellon Steven U Walkley Douglas F Covey Jean E Schaffer Daniel S Ory

Niemann-Pick type C1 (NPC1) disease is a fatal neurodegenerative disease characterized by neuronal lipid storage and progressive Purkinje cell loss in the cerebellum. We investigated whether therapeutic approaches to bypass the cholesterol trafficking defect in NPC1 disease might delay disease progression in the npc1(-/-) mouse model. We show that the neurosteroid allopregnanolone (ALLO) and T0...

2017
Sandra Torres Nuria Matías Anna Baulies Susana Nuñez Cristina Alarcon-Vila Laura Martinez Natalia Nuño Anna Fernandez Joan Caballeria Thierry Levade Alba Gonzalez-Franquesa Pablo Garcia-Rovés Elisa Balboa Silvana Zanlungo Gemma Fabrías Josefina Casas Carlos Enrich Carmen Garcia-Ruiz José C. Fernández-Checa

Niemann Pick type C (NPC) disease is a progressive lysosomal storage disorder caused by mutations in genes encoding NPC1/NPC2 proteins, characterized by neurological defects, hepatosplenomegaly and premature death. While the primary biochemical feature of NPC disease is the intracellular accumulation of cholesterol and gangliosides, predominantly in endolysosomes, mitochondrial cholesterol accu...

2014
Masashi Kamikawa XiaoFeng Lei Yukio Fujiwara Kazuchika Nishitsuji Hiroshi Mizuta Motohiro Takeya Naomi Sakashita

We previously demonstrated that macrophages exhibit endoplasmic reticulum fragmentation under cholesterol-rich conditions, which results in the generation of acyl-coenzyme A: cholesterol acyltransferase 1 (ACAT1)-associated late endosomes/lysosomes (ACAT1-LE). ACAT1-LE efficiently esterify free cholesterol in loco, even with abnormal egress of free cholesterol from late endosomes. Because impai...

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