(Q48120696)

8 February 2018

title

Lysosomal enzyme replacement of the brain with intravenous non-viral gene transfer (English)

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8 February 2018

author

William M Pardridge

4

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8 February 2018

Ruben J Boado

3

object named as

Ruben J Boado

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8 February 2018

author name string

Yun Zhang

1

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8 February 2018

Yuntao Wang

2

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8 February 2018

publication date

30 June 2007

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8 February 2018

published in

Pharmaceutical Research

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8 February 2018

volume

25

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8 February 2018

issue

2

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8 February 2018

page(s)

400-406

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8 February 2018

Intractable fever and cortical neuronal glycogen storage in glycogenosis type 2.

cites work

1 reference

Crossref

https://api.crossref.org/works/10.1007%2FS11095-007-9357-6

Receptor-mediated gene targeting to tissues in vivo following intravenous administration of pegylated immunoliposomes

21 January 2018

1 reference

12 December 2020

Adeno-associated virus vector-mediated transduction in the cat brain

1 reference

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Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer.

1 reference

12 December 2020

Characterization and distribution of transferrin receptors in the rat brain.

1 reference

12 December 2020

Site-directed mutagenesis of cysteine residues of large neutral amino acid transporter LAT1.

1 reference

12 December 2020

High level expression and export of beta-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector.

1 reference

12 December 2020

Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesions in a large volume of brain in mucopolysaccharidosis VII mice.

1 reference

12 December 2020

Decline in exogenous gene expression in primate brain following intravenous administration is due to plasmid degradation.

1 reference

12 December 2020

Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector

1 reference

12 December 2020

Brain insulin receptors and spatial memory. Correlated changes in gene expression, tyrosine phosphorylation, and signaling molecules in the hippocampus of water maze trained rats

1 reference

12 December 2020

Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency

1 reference

12 December 2020

Gene targeting in vivo with pegylated immunoliposomes

1 reference

12 December 2020

Genetic correction of the fetal brain increases the lifespan of mice with the severe multisystemic disease mucopolysaccharidosis type VII.

1 reference

12 December 2020

Active site residues of human beta-glucuronidase. Evidence for Glu(540) as the nucleophile and Glu(451) as the acid-base residue

1 reference

12 December 2020

Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors

1 reference

12 December 2020

Overexpression rescues the mutant phenotype of L176F mutation causing beta-glucuronidase deficiency mucopolysaccharidosis in two Mennonite siblings.

1 reference

12 December 2020

Drug and gene delivery to the brain: the vascular route

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12 December 2020

Brain-specific expression of an exogenous gene after i.v. administration

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12 December 2020

Targeted transgene expression in rat brain using lentiviral vectors.

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12 December 2020

Marked enhancement in gene expression by targeting the human insulin receptor

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12 December 2020

Global non-viral gene transfer to the primate brain following intravenous administration.

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12 December 2020

C-terminal processing of human beta-glucuronidase. The propeptide is required for full expression of catalytic activity, intracellular retention, and proper phosphorylation.

1 reference

12 December 2020

Evaluation of pathological manifestations of disease in mucopolysaccharidosis VII mice after neonatal hepatic gene therapy.

1 reference

12 December 2020

Selective expression of the large neutral amino acid transporter at the blood-brain barrier

1 reference

12 December 2020

Noninvasive gene targeting to the brain

1 reference

12 December 2020

Targeting of an anti-CR3 (CD11b/CD18) monoclonal antibody to spleen but not brain, in vivo in mice.

1 reference

12 December 2020

Widespread gene delivery and structure-specific patterns of expression in the brain after intraventricular injections of neonatal mice with an adeno-associated virus vector

1 reference

12 December 2020

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.

1 reference

12 December 2020

Gene therapy for lysosomal storage disorders with neuropathology

1 reference

12 December 2020

Stable levels of long-term transgene expression driven by the latency-associated transcript promoter in a herpes simplex virus type 1 vector

1 reference

12 December 2020

Correction of murine mucopolysaccharidosis VII by a human beta-glucuronidase transgene

1 reference

12 December 2020

Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier

1 reference

12 December 2020

Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors.

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12 December 2020

10.1007/S11095-007-9357-6

Identifiers

DOI

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8 February 2018

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8 February 2018