Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer.
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New Strategies for the Treatment of Phenylketonuria (PKU)Phenylketonuria: translating research into novel therapiesDifferential effects of low-phenylalanine protein sources on brain neurotransmitters and behavior in C57Bl/6-Pah(enu2) miceThe Behavioral Consequence of Phenylketonuria in Mice Depends on the Genetic BackgroundComplete correction of hyperphenylalaninemia following liver-directed, recombinant AAV2/8 vector-mediated gene therapy in murine phenylketonuria.Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver PromoterAAV2/8-mediated correction of OTC deficiency is robust in adult but not neonatal Spf(ash) mice.Intensive pharmacological immunosuppression allows for repetitive liver gene transfer with recombinant adenovirus in nonhuman primatesComparison of adeno-associated virus pseudotype 1, 2, and 8 vectors administered by intramuscular injection in the treatment of murine phenylketonuriaIndirect action of tumor necrosis factor-alpha in liver injury during the CD8+ T cell response to an adeno-associated virus vector in mice.Phenotypic reversion of fair hair upon gene therapy of the phenylketonuria mice.Phenylalanine hydroxylase deficiency.A sensitive assay system to test antisense oligonucleotides for splice suppression therapy in the mouse liver.Phenylalanine hydroxylase misfolding and pharmacological chaperones.A potential role of distinctively delayed blood clearance of recombinant adeno-associated virus serotype 9 in robust cardiac transduction.Up to date knowledge on different treatment strategies for phenylketonuria.Treatment of phenylketonuria using minicircle-based naked-DNA gene transfer to murine liver.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.Potential of AAV vectors in the treatment of metabolic disease.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.Hepatocyte transplantation improves phenotype and extends survival in a murine model of intermediate maple syrup urine disease.AAV gene therapy corrects OTC deficiency and prevents liver fibrosis in aged OTC-knock out heterozygous mice.Effects of transient immunosuppression on adenoassociated, virus-mediated, liver-directed gene transfer in rhesus macaques and implications for human gene therapy.Long-term cardiac-targeted RNA interference for the treatment of heart failure restores cardiac function and reduces pathological hypertrophy.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.Improved Lentiviral Gene Delivery to Mouse Liver by Hydrodynamic Vector Injection through Tail Vein
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P2860
Administration-route and gender-independent long-term therapeutic correction of phenylketonuria (PKU) in a mouse model by recombinant adeno-associated virus 8 pseudotyped vector-mediated gene transfer.
description
2006 nî lūn-bûn
@nan
2006年の論文
@ja
2006年学术文章
@wuu
2006年学术文章
@zh-cn
2006年学术文章
@zh-hans
2006年学术文章
@zh-my
2006年学术文章
@zh-sg
2006年學術文章
@yue
2006年學術文章
@zh
2006年學術文章
@zh-hant
name
Administration-route and gende ...... vector-mediated gene transfer.
@en
Administration-route and gende ...... correction of phenylketonuria
@nl
type
label
Administration-route and gende ...... vector-mediated gene transfer.
@en
Administration-route and gende ...... correction of phenylketonuria
@nl
prefLabel
Administration-route and gende ...... vector-mediated gene transfer.
@en
Administration-route and gende ...... correction of phenylketonuria
@nl
P2093
P2860
P356
P1433
P1476
Administration-route and gende ...... vector-mediated gene transfer.
@en
P2093
P2860
P2888
P304
P356
10.1038/SJ.GT.3302684
P577
2006-04-01T00:00:00Z
P5875
P6179
1020856348