(Q95780950)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28182871%20AND%20SRC:MED&resulttype=core&format=json

30 May 2020

title

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product (English)

1 reference

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30 May 2020

5

Bruce R Blazar

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28182871%20AND%20SRC:MED&resulttype=core&format=json

30 May 2020

author name string

Mark J Osborn

1

1 reference

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30 May 2020

Ron T McElmurry

2

1 reference

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30 May 2020

Brandon Peacock

3

1 reference

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30 May 2020

Jakub Tolar

4

1 reference

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30 May 2020

publication date

1 August 2008

1 reference

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30 May 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28182871%20AND%20SRC:MED&resulttype=core&format=json

30 May 2020

volume

16

1 reference

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30 May 2020

issue

8

1 reference

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30 May 2020

page(s)

1459-1466

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28182871%20AND%20SRC:MED&resulttype=core&format=json

Cervical myelopathy due to dural compression in mucopolysaccharidosis

30 May 2020

cites work

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Cervical cord compression in mucopolysaccharidosis

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Spinal pachymeningitis secondary to mucopolysaccharidosis. Case report.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Cerebrospinal fluid shunts in the management of behavioural problems in Sanfilippo syndrome (MPS III).

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Enzyme replacement therapy for the mucopolysaccharide storage disorders

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Microglia: The Effector Cell for Reconstitution of the Central Nervous System following Bone Marrow Transplantation for Lysosomal and Peroxisomal Storage Diseases

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Outcome after three years of laronidase enzyme replacement therapy in a patient with Hurler syndrome

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Enzyme replacement therapy in two patients with an advanced severe (Hurler) phenotype of mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Outwitting the blood-brain barrier for therapeutic purposes: osmotic opening and other means

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

The blood-brain barrier: bottleneck in brain drug development

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Targeted delivery of proteins across the blood-brain barrier

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Delivery of beta-galactosidase to mouse brain via the blood-brain barrier transferrin receptor

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

A picornaviral 2A-like sequence-based tricistronic vector allowing for high-level therapeutic gene expression coupled to a dual-reporter system

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Capillary depletion method for quantification of blood-brain barrier transport of circulating peptides and plasma proteins

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Molecular anatomy of the brain endothelial barrier: an overview of the distributional features

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Lipoprotein receptor binding, cellular uptake, and lysosomal delivery of fusions between the receptor-associated protein (RAP) and alpha-L-iduronidase or acid alpha-glucosidase

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Recombinant granulocyte colony-stimulating factor-transferrin fusion protein as an oral myelopoietic agent

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Gene transfer vector biodistribution: pivotal safety studies in clinical gene therapy development

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Biodistribution and toxicity studies of VSVG-pseudotyped lentiviral vector after intravenous administration in mice with the observation of in vivo transduction of bone marrow

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Gene therapy of storage disorders by retroviral and lentiviral vectors

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Epinephrine enhances lysosomal enzyme delivery across the blood brain barrier by up-regulation of the mannose 6-phosphate receptor

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Limited transgene immune response and long-term expression of human alpha-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Intrathecal administration of AAV vectors for the treatment of lysosomal storage in the brains of MPS I mice

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

A fluorometric assay using 4-methylumbelliferyl α-l-iduronide for the estimation of α-l-iduronidase activity and the detection of Hurler and Scheie syndromes

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice

1 reference

https://api.crossref.org/works/10.1038%2FMT.2008.119

7 January 2021

Identifiers

DOI

10.1038/MT.2008.119

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28182871%20AND%20SRC:MED&resulttype=core&format=json

30 May 2020

1 reference