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1 GNE (UDP-GlcNAc 2-epimerase/ManNAc kinase) myopathy is a
2 GNE deficiency may affect levels of UDP-GlcNAc, a key me
3 GNE harbors two enzymatic activities required for biosyn
4 GNE myopathy is a rare recessive myopathy associated wit
5 GNE Myopathy is a rare recessively inherited neuromuscul
6 GNE myopathy is an autosomal recessive muscle disease ca
7 GNE myopathy is an important diagnosis to consider in pa
8 GNE-6640 and GNE-6776 interact with acidic residues that
9 GNE-KLH (keyhole limpet hemocyannin) was found to elicit
12 is effort culminated in the discovery of 20 (GNE-555), a highly potent, selective, metabolically stab
18 caine (dAd5GNE), based on the cocaine analog GNE linked to the capsid proteins of a serotype 5 adenov
19 he rapid identification of GNE-0877 (11) and GNE-9605 (20) as highly potent and selective LRRK2 inhib
21 Structural studies reveal that GNE-6640 and GNE-6776 non-covalently target USP7 12 A distant from th
23 d with HMN/CMT2 (ARHGEF28, KBTBD13, AGRN and GNE); in genes previously associated with HMN/CMT2 but i
25 scovery of small molecule inhibitors such as GNE-7915 (18) and 19, which possess an ideal balance of
29 The most advanced delta-sultam compound, GNE-3500 (27, 1-{4-[3-fluoro-4-((3S,6R)-3-methyl-1,1-dio
30 f these, GNE-235, and its enantiomer control GNE-234 are suggested for initial cellular investigation
31 c siRNAs specifically targeting the dominant GNE mutation c.797G>A (p.R266Q) in sialuria fibroblasts.
33 s S-nitrosylation, accounts for the enhanced GNE that we have observed and that has been previously o
35 O2 mediates Ras guanine nucleotide exchange (GNE) by conversion of Ras-bound GDP into an unstable 5-n
36 hown to promote guanine nucleotide exchange (GNE) on Ras and increase cellular Ras-GTP levels, but th
38 xpanded; GE) and without (gene-not-expanded; GNE) the trinucleotide cytosine, adenine, guanine (CAG)
44 tion to the three previously described human GNE isoforms (hGNE1-hGNE3), our database and polymerase
46 unctional and regulatory mechanisms of human GNE and may contribute to further elucidating the pathol
47 izing disease-causing mutations in the human GNE gene that give rise to sialuria, hereditary inclusio
48 hology and treatment strategies of the human GNE-opathies sialuria and hereditary inclusion body myop
50 ng to galectin-1, indicating that changes in GNE activity can alter affinity of cell-surface glycopro
54 cle disease caused by biallelic mutations in GNE, a gene encoding for a single protein with key enzym
57 G2019S LCLs with the LRRK2 kinase inhibitor GNE-7915, either prevented or restored mtDNA damage to c
60 X015 or pelabresib or the CBP/p300 inhibitor GNE-049 was synergistically lethal in MPN-sAML cells (in
61 identification of type II pan-RAF inhibitor GNE-0749 (7), which features a fluoroquinazolinone hinge
62 ion of a potent and selective ITK inhibitor (GNE-9822) with good ADME properties in preclinical speci
63 e MAP4K4-specific pharmacological inhibitor, GNE-495, impedes pancreatic cancer cell growth, migratio
65 for UDP-GlcNAc 2-epimerase/ManNAc 6-kinase (GNE) beyond controlling flux into the sialic acid biosyn
67 imerase/N-acetylmannosamine (ManNAc) kinase (GNE/MNK), result in hereditary inclusion body myopathy (
68 deposition of maternal gne mRNA and maternal GNE protein at the earliest embryonic stage, emphasizing
72 To investigate other potential effects of GNE mutations, we compared sialic acid production in cel
73 s GM3 and GD3 also changed the expression of GNE and led to reduced ST3Gal5 and ST8Sia1 mRNA levels,
76 rs culminated in the rapid identification of GNE-0877 (11) and GNE-9605 (20) as highly potent and sel
77 The resultant loss of feedback inhibition of GNE-epimerase activity by CMP-sialic acid causes excessi
81 missense mutations in the allosteric site of GNE, coding for the rate-limiting enzyme of sialic acid
82 alic acid supplementation on symptomatic old GNE myopathy mice that have ongoing, active muscle degen
86 50 years) with genetically and biopsy-proved GNE-related myopathy and five healthy volunteers (three
87 y proposed a mechanism of NO/O2-mediated Ras GNE, in which *NO2, formed by the reaction of NO with O2
90 thiyl radical intermediate that promotes Ras GNE, we have also postulated that another byproduct of t
91 creased ganglioside levels (e.g. recombinant GNE and exogenous gangliosides) led to reduced prolifera
93 We propose one example from this series, GNE-064, as a chemical probe for the bromodomains SMARCA
94 ion to exhibiting low levels of sialylation, GNE-deficient cells produced distinct N-linked glycan st
96 to a third-generation cocaine hapten, termed GNE (6-(2R,3S)-3-(benzoyloxy)-8-methyl-8-azabicyclo [3.2
101 and mass spectrometry analysis revealed that GNE deficiency is associated with unanticipated effects
104 muscular disorder caused by mutations in the GNE gene, which codes for the key enzyme in the metaboli
111 enzyme for sialic acid biosynthesis, lead to GNE myopathy, a disease manifesting with progressive mus
117 suggest an unanticipated mechanism by which GNE activity might affect signaling through cell-surface
124 SWV was significantly lower in patients with GNE-related myopathy than in control subjects (P < .02).
125 muscles were evaluated in the patients with GNE-related myopathy, and the gastrocnemius, vastus late