نتایج جستجو برای: gne

تعداد نتایج: 282  

2008
Chris Jay Gregory Nemunaitis John Nemunaitis Neil Senzer Stephan Hinderlich Daniel Darvish Julie Ogden John Eager Alex Tong Phillip B Maples

Hereditary Inclusion Body Myopathy (HIBM2) is a chronic progressive skeletal muscle wasting disorder which generally leads to complete disability before the age of 50 years. There is currently no effective therapeutic treatment for HIBM2. Development of this disease is related to expression in family members of an autosomal recessive mutation of the GNE gene, which encodes the bifunctional enzy...

2011
Irit Milman Krentsis Ilan Sela Rachel Eiges Véronique Blanchard Markus Berger Michal Becker Cohen Stella Mitrani-Rosenbaum

UDP-N-acetylglucosamine 2 epimerase/N-acetylmannosamime kinase (GNE) is a bifunctional enzyme which catalyzes the two key sequential steps in the biosynthetic pathway of sialic acid, the most abundant terminal monosaccharide on glycoconjugates of eukaryotic cells. GNE knock out (GNE KO) mice are embryonically lethal at day E8.5. Although the role of GNE in the sialic pathway has been well estab...

2017
Nam D Pham Poh-Choo Pang Soumya Krishnamurthy Amberlyn M Wands Paola Grassi Anne Dell Stuart M Haslam Jennifer J Kohler

GNE (UDP-GlcNAc 2-epimerase/ManNAc kinase) myopathy is a rare muscle disorder associated with aging and is related to sporadic inclusion body myositis, the most common acquired muscle disease of aging. Although the cause of sporadic inclusion body myositis is unknown, GNE myopathy is associated with mutations in GNE. GNE harbors two enzymatic activities required for biosynthesis of sialic acid ...

2017
Jennifer Garland Joshi Stephen Bradley Class Angela Gruber Carla Ciccone Aaron Poliak Christina P Hayes Vandana Singhal Christina Slota John Perreault Ralitza Gavrilova Joseph A Shrader Prashant Chittiboina Galen Joe John Heiss William A Gahl Marjan Huizing Nuria Carrillo May Christine V Malicdan

BACKGROUND GNE myopathy is a rare genetic disease characterized by progressive muscle atrophy and weakness. It is caused by biallelic mutations in the GNE gene that encodes for the bifunctional enzyme, uridine diphosphate (UDP)-N-acetylglucosamine (GlcNAc) 2-epimerase/N-acetylmannosamine (ManNAc) kinase. Typical characteristics of GNE myopathy include progressive myopathy, first involving anter...

2010
Thomas Paccalet Zoé Coulombe Jacques P. Tremblay

OBJECTIVE HIBM (Hereditary Inclusion Body Myopathy) is a recessive hereditary disease characterized by adult-onset, slowly progressive muscle weakness sparing the quadriceps. It is caused by a single missense mutation of each allele of the UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase (GNE) gene, a bifunctional enzyme catalyzing the first two steps of sialic acid synthesis in m...

Journal: :Journal of medical and dental sciences 2008
Shoichiro Ishihara Hiroyuki Tomimitsu Hiroto Fujigasaki Fumiaki Saito Hidehiro Mizusawa

BACKGROUND UDP-N-acetylglucosamine 2-epimerase/N-acetylmannosamine kinase (GNE) is a key molecule in the pathogenesis of distal myopathy with rimmed vacuoles (DMRV) and hereditary inclusion body myopathy (HIBM) and almost all such patients have some mutations in GNE. However, subcellular localization of GNE and the mechanism of muscular damage have not been clarified. METHODS A rabbit polyclo...

Journal: :The Journal of clinical investigation 2007
Belinda Galeano Riko Klootwijk Irini Manoli MaoSen Sun Carla Ciccone Daniel Darvish Matthew F Starost Patricia M Zerfas Victoria J Hoffmann Shelley Hoogstraten-Miller Donna M Krasnewich William A Gahl Marjan Huizing

Mutations in the key enzyme of sialic acid biosynthesis, uridine diphospho-N-acetylglucosamine 2-epimerase/N-acetylmannosamine (ManNAc) kinase (GNE/MNK), result in hereditary inclusion body myopathy (HIBM), an adult-onset, progressive neuromuscular disorder. We created knockin mice harboring the M712T Gne/Mnk mutation. Homozygous mutant (Gne(M712T/M712T)) mice did not survive beyond P3. At P2, ...

2015
Mathieu Cerino Svetlana Gorokhova Anthony Béhin Jon Andoni Urtizberea Virginie Kergourlay Eric Salvo Rafaëlle Bernard Nicolas Lévy Marc Bartoli Martin Krahn

BACKGROUND GNE myopathy is a rare autosomal recessively inherited muscle disease resulting from mutations in the gene encoding GNE (UDP-N-acetylglucosamine-2-epimerase/N-acetylmannosamine kinase), a key enzyme in sialic acid biosynthesis. 154 different pathogenic variants have been previously associated with GNE myopathy. OBJECTIVE Describe novel pathogenic variants associated with GNE myopat...

Journal: :Human molecular genetics 2014
Alon Daya Gad David Vatine Michal Becker-Cohen Tzukit Tal-Goldberg Adam Friedmann Yoav Gothilf Shao Jun Du Stella Mitrani-Rosenbaum

GNE Myopathy is a rare recessively inherited neuromuscular disorder caused by mutations in the GNE gene, which codes for the key enzyme in the metabolic pathway of sialic acid synthesis. The process by which GNE mutations lead to myopathy is not well understood. By in situ hybridization and gne promoter-driven fluorescent transgenic fish generation, we have characterized the spatiotemporal expr...

Journal: :PLoS ONE 2008
Shira Amsili Hagit Zer Stephan Hinderlich Sabine Krause Michal Becker-Cohen Daniel G. MacArthur Kathryn N. North Stella Mitrani-Rosenbaum

BACKGROUND Hereditary inclusion body myopathy (HIBM) is a rare neuromuscular disorder caused by mutations in GNE, the key enzyme in the biosynthetic pathway of sialic acid. While the mechanism leading from GNE mutations to the HIBM phenotype is not yet understood, we searched for proteins potentially interacting with GNE, which could give some insights about novel putative biological functions ...

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