Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.
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Phenylketonuria: translating research into novel therapiesCurative ex vivo liver-directed gene therapy in a pig model of hereditary tyrosinemia type 1.Comparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuriaThe role of hepatocyte hemojuvelin in the regulation of bone morphogenic protein-6 and hepcidin expression in vivo.Low bone strength is a manifestation of phenylketonuria in mice and is attenuated by a glycomacropeptide diet.Phenylketonuria: an inborn error of phenylalanine metabolism.Up to date knowledge on different treatment strategies for phenylketonuria.Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.Glycomacropeptide is a prebiotic that reduces Desulfovibrio bacteria, increases cecal short-chain fatty acids, and is anti-inflammatory in mice.Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis.Glycomacropeptide, a low-phenylalanine protein isolated from cheese whey, supports growth and attenuates metabolic stress in the murine model of phenylketonuria.Protective effect of recombinant adeno-associated virus 2/8-mediated gene therapy from the maternal hyperphenylalaninemia in offsprings of a mouse model of phenylketonuria.Gene Therapy for the Treatment of Neurological Disorders: Metabolic Disorders.Progress toward cell-directed therapy for phenylketonuria.Correction of murine PKU following AAV-mediated intramuscular expression of a complete phenylalanine hydroxylating systemGene therapy for liver enzyme deficiencies: what have we learned from models for Crigler-Najjar and tyrosinemia?Clinical therapeutics for phenylketonuria.Noninvasive 3-dimensional imaging of liver regeneration in a mouse model of hereditary tyrosinemia type 1 using the sodium iodide symporter gene.Construction and application of a novel hepatocyte-directed vector to simultaneous knockdown and overexpression of multiple genes.Complete restoration of phenylalanine oxidation in phenylketonuria mouse by a self-complementary adeno-associated virus vector.Phenylketonuria: A new look at an old topic, advances in laboratory diagnosis, and therapeutic strategies.In search of proof-of-concept: gene therapy for glycogen storage disease type Ia.Blood phenylalanine reduction corrects CNS dopamine and serotonin deficiencies and partially improves behavioral performance in adult phenylketonuric mice.
P2860
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P2860
Complete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.
description
2006 nî lūn-bûn
@nan
2006 թուականի Մարտին հրատարակուած գիտական յօդուած
@hyw
2006 թվականի մարտին հրատարակված գիտական հոդված
@hy
2006年の論文
@ja
2006年論文
@yue
2006年論文
@zh-hant
2006年論文
@zh-hk
2006年論文
@zh-mo
2006年論文
@zh-tw
2006年论文
@wuu
name
Complete correction of hyperph ...... apy in murine phenylketonuria.
@ast
Complete correction of hyperph ...... apy in murine phenylketonuria.
@en
type
label
Complete correction of hyperph ...... apy in murine phenylketonuria.
@ast
Complete correction of hyperph ...... apy in murine phenylketonuria.
@en
prefLabel
Complete correction of hyperph ...... apy in murine phenylketonuria.
@ast
Complete correction of hyperph ...... apy in murine phenylketonuria.
@en
P2093
P2860
P356
P1433
P1476
Complete correction of hyperph ...... rapy in murine phenylketonuria
@en
P2093
C O Harding
D D Koeberl
E Goebel-Daghighi
P2860
P2888
P304
P356
10.1038/SJ.GT.3302678
P577
2006-03-01T00:00:00Z