نتایج جستجو برای: lysosomal storage disease

تعداد نتایج: 1671181  

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 ...

2015
Markus Damme Stijn Stroobants Meike Lüdemann Michelle Rothaug Renate Lüllmann‐Rauch Hans Christian Beck Annika Ericsson Claes Andersson Jens Fogh Rudi D'Hooge Paul Saftig Judith Blanz

OBJECTIVE The lysosomal storage disease alpha-mannosidosis is caused by the deficiency of the lysosomal acid hydrolase alpha-mannosidase (LAMAN) leading to lysosomal accumulation of neutral mannose-linked oligosaccharides throughout the body, including the brain. Clinical findings in alpha-mannosidosis include skeletal malformations, intellectual disabilities and hearing impairment. To date, no...

2011
Sayali S. Dixit Michel Jadot Istvan Sohar David E. Sleat Ann M. Stock Peter Lobel

Niemann-Pick Type C (NPC) disease is a lysosomal storage disorder characterized by accumulation of unesterified cholesterol and other lipids in the endolysosomal system. NPC disease results from a defect in either of two distinct cholesterol-binding proteins: a transmembrane protein, NPC1, and a small soluble protein, NPC2. NPC1 and NPC2 are thought to function closely in the export of lysosoma...

Journal: :Proceedings of the National Academy of Sciences of the United States of America 1999
M Jeyakumar T D Butters M Cortina-Borja V Hunnam R L Proia V H Perry R A Dwek F M Platt

Sandhoff disease is a neurodegenerative disorder resulting from the autosomal recessive inheritance of mutations in the HEXB gene, which encodes the beta-subunit of beta-hexosaminidase. GM2 ganglioside fails to be degraded and accumulates within lysosomes in cells of the periphery and the central nervous system (CNS). There are currently no therapies for the glycosphingolipid lysosomal storage ...

Journal: :International Journal of Community Medicine and Public Health 2023

Nieman-Pick disease (NPD) also known as acid sphingomyelinase deficiency (ASMD) is a rare autosomal recessive disorder of lysosomal storage which affects 1 in 250,000 individuals mainly belonging to the Ashkenazi Jew population. The prevalence varies across three main subtypes where type A and B 40,000 C 150,000 individuals, being more prevalent French-Acadian descent region Nova Scotia.

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