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1 PKU mice were impaired on both odor discrimination rever
2 PKU mice were impaired on reversals 2, 3, and 4.
3 PKU produces cognitive deficits in humans if it is untre
5 nt work, we generate a more potent EcN-based PKU strain through optimization of whole cell PAL activi
7 global phenotype distribution of 62% classic PKU, 22% mild PKU, and 16% mild hyperphenylalaninemia.
9 tribution exists across Europe, from classic PKU in the east to mild PKU in the southwest and mild hy
12 d metabolic camp intervention and to compare PKU profiles with those of matched healthy controls.
13 Phenylalanine hydroxylase (Pah)-deficient "PKU mice" have a mutation in the Pah gene that causes ph
14 he identification of two metabolic diseases (PKU and tyrosinemia) through blood analysis with minimal
15 find the water faster on day 2, pre-exposed PKU mice showed no significant benefit from their pre-ex
20 that globally 0.45 million individuals have PKU, with global prevalence 1:23,930 live births (range
23 -edited hepatocyte cell line and a humanized PKU mouse model, we demonstrate efficient in vitro and i
24 sing lipid nanoparticles (LNPs) in humanized PKU mice, we observe complete and durable normalization
28 f patients with different IEM (especially in PKU and MPS patients, but also in IEM under protein-rest
31 of hyperphenylalanemia on liver functions in PKU mice was assessed by transcriptome and proteomic ana
32 ons of L-Phe were nearly six-fold greater in PKU mice (863.12 +/- 17.96 micromol/kg) than in their he
33 omplete normalization of blood Phe levels in PKU mice, with up to 52% whole-liver corrective PAH edit
38 individuals aged 0-12 years and for maternal PKU, and 120-600 mumol/L for non-pregnant individuals ol
39 to normal or above-normal values in maternal PKU pregnancies for a period of > or = 3 h by supplement
40 mino acid profile in a rat model of maternal PKU induced by DL-alpha-methylphenylalanine (AMPhe).
42 g to conceive and during pregnancy (maternal PKU), untreated phenylalanine blood concentrations of mo
44 Europe, from classic PKU in the east to mild PKU in the southwest and mild hyperphenylalaninemia in t
45 ority of patients (usually those with milder PKU), and pegylated phenylalanine ammonia lyase, which r
46 pite dietary and medical interventions, most PKU individuals harboring at least one c.1222C>T variant
47 3 umol/L (IQR: -325, -28 umol/L)] and 70% of PKU participants demonstrated improved dietary adherence
50 nylalanine hydroxylase cDNA to the livers of PKU mice by hydrodynamic injection of plasmid DNA and sh
52 rst evidence that the genetic mouse model of PKU (Pah(enu2)) also produces cognitive impairments.
54 d PAH P281L and ABCC6 R1164X mouse models of PKU and PXE, we show that when formulated in lipid nanop
56 r as a potential option for the treatment of PKU patients or as a possible model for the substitutive
58 We show that young patients with MSUD or PKU have decreased average RBC UDPgalactose concentratio
60 lthy participants, phenylketonuric patients (PKU), patients with mucopolysaccharidoses (MPS), patient
77 ations in this enzyme cause phenylketonuria (PKU), a genetic disorder that leads to brain damage and
78 ydroxylase gene (PAH) cause phenylketonuria (PKU), PAH was studied for normal polymorphisms and linka
85 aired brain function during phenylketonuria (PKU), the most common biochemical cause of mental retard
90 rat model in which maternal phenylketonuria (PKU) is induced by the inclusion of an inhibitor of phen
92 syrup urine disease (MSUD), phenylketonuria (PKU), and other metabolic diseases who were treated with
97 ice exhibit phenotypes that closely resemble PKU patients such as retarded body growth, cutaneous hyp
99 a CoQ values were significantly lower in the PKU and MPS groups than in controls and neurological pat
100 n of L-Phe at concentrations observed in the PKU brain depresses glutamatergic synaptic transmission
101 epressant effects of L-Phe take place in the PKU brain, then chronic impairment of the glutamate syst
102 s study aimed to characterize changes in the PKU plasma metabolome following a 5-d metabolic camp int
103 We sought to characterize the impact of the PKU genotype and dietary protein source on bone biomecha
108 s, serum phenylalanine levels in all treated PKU mice were reduced to the normal range and remained s
111 oncentrated on European populations in which PKU was common and showed that haplotype-frequency varia
113 ed fatty acids were elevated in females with PKU at baseline, whereas fatty acylcholine metabolites w
115 umans, brain dysfunction in individuals with PKU is still not well understood and further research is
118 he management and follow-up of patients with PKU are scheduled according to age, adherence to treatme
124 layed development in offspring of women with PKU is associated with lack of maternal metabolic contro