Gene redundancy and pharmacological gene therapy: implications for X-linked adrenoleukodystrophy. (Q38331491)

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6 December 2019

title

Gene redundancy and pharmacological gene therapy: implications for X-linked adrenoleukodystrophy (English)

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6 December 2019

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1

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6 December 2019

author name string

H M Wei

2

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6 December 2019

J F Lu

3

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6 December 2019

L T Braiterman

4

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6 December 2019

M C McGuinness

5

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A B Moser

6

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P A Watkins

7

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6 December 2019

K D Smith

8

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6 December 2019

language of work or name

English

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publication date

1 November 1998

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6 December 2019

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6 December 2019

volume

4

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6 December 2019

issue

11

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6 December 2019

page(s)

1261-1268

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https://scigraph.springernature.com/pub.10.1038/3242

6 December 2019

exact match

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cites work

Evolutionary origins and maintenance of redundant gene expression during metazoan development

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7 January 2021

Thinking about genetic redundancy

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Treatment of sickle cell anemia with 5-azacytidine results in increased fetal hemoglobin production and is associated with nonrandom hypomethylation of DNA around the gamma-delta-beta-globin gene complex

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A short-term trial of butyrate to stimulate fetal-globin-gene expression in the beta-globin disorders

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Amelioration of the dystrophic phenotype of mdx mice using a truncated utrophin transgene

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Backup gene may help muscles help themselves

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Direct demonstration that the deficient oxidation of very long chain fatty acids in X-linked adrenoleukodystrophy is due to an impaired ability of peroxisomes to activate very long chain fatty acids

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7 January 2021

Adrenoleukodystrophy: impaired oxidation of fatty acids due to peroxisomal lignoceroyl-CoA ligase deficiency

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7 January 2021

Putative X-linked adrenoleukodystrophy gene shares unexpected homology with ABC transporters

1 reference

7 January 2021

Identification of a two base pair deletion in five unrelated families with adrenoleukodystrophy: a possible hot spot for mutations.

1 reference

7 January 2021

Mutational analysis of patients with X-linked adrenoleukodystrophy

1 reference

7 January 2021

The gene responsible for adrenoleukodystrophy encodes a peroxisomal membrane protein

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7 January 2021

Identification of mutations in the ALD-gene of 20 families with adrenoleukodystrophy/adrenomyeloneuropathy.

1 reference

7 January 2021

Retroviral-mediated gene transfer corrects very-long-chain fatty acid metabolism in adrenoleukodystrophy fibroblasts

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7 January 2021

Suppression of peroxisomal membrane protein defects by peroxisomal ATP binding cassette (ABC) proteins

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7 January 2021

A close relative of the adrenoleukodystrophy (ALD) gene codes for a peroxisomal protein with a specific expression pattern

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7 January 2021

cDNA cloning and mRNA expression of the human adrenoleukodystrophy related protein (ALDRP), a peroxisomal ABC transporter.

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Mutations in the 70K peroxisomal membrane protein gene in Zellweger syndrome

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Identification of a fourth half ABC transporter in the human peroxisomal membrane

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Primary structure of human PMP69, a putative peroxisomal ABC-transporter

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Clinical variation in X-linked adrenoleukodystrophy: fatty acid and lipid metabolism in cultured fibroblasts

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A mouse model for X-linked adrenoleukodystrophy

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Adrenoleukodystrophy: phenotype, genetics, pathogenesis and therapy

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Very long chain fatty acids in X-linked adrenoleukodystrophy brain after treatment with Lorenzo's oil.

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7 January 2021

Brain, liver, and adipose tissue erucic and very long chain fatty acid levels in adrenoleukodystrophy patients treated with glyceryl trierucate and trioleate oils (Lorenzo's oil).

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7 January 2021

Long-term treatment of girls with ornithine transcarbamylase deficiency

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7 January 2021

Molecular and functional characterization of the murine glucocerebrosidase gene

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Two cytotoxic cell proteinase genes are differentially sensitive to sodium butyrate

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7 January 2021

Transcriptional upregulation of TGF-alpha by phenylacetate and phenylbutyrate is associated with differentiation of human melanoma cells

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7 January 2021

Fenofibrate differently alters expression of genes encoding ATP-binding transporter proteins of the peroxisomal membrane

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7 January 2021

In vitro pharmacologic restoration of CFTR-mediated chloride transport with sodium 4-phenylbutyrate in cystic fibrosis epithelial cells containing delta F508-CFTR.

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A pilot clinical trial of oral sodium 4-phenylbutyrate (Buphenyl) in deltaF508-homozygous cystic fibrosis patients: partial restoration of nasal epithelial CFTR function

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ALDP expression in fibroblasts of patients with X-linked adrenoleukodystrophy

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7 January 2021

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Peroxisomal acyl-coenzyme A oxidase is a rate-limiting enzyme in a very-long-chain fatty acid beta-oxidation system

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7 January 2021

Expression of the adrenoleukodystrophy protein in the human and mouse central nervous system

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7 January 2021

Antibodies directed against the peroxisomal targeting signal of firefly luciferase recognize multiple mammalian peroxisomal proteins

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7 January 2021

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The structure of the human sterol carrier protein X/sterol carrier protein 2 gene (SCP2).

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The Candida boidinii peroxisomal membrane protein Pmp30 has a role in peroxisomal proliferation and is functionally homologous to Pmp27 from Saccharomyces cerevisiae

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7 January 2021

Giant peroxisomes in oleic acid-induced Saccharomyces cerevisiae lacking the peroxisomal membrane protein Pmp27p

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7 January 2021

Peroxisomal fatty acid beta-oxidation in HepG2 cells

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7 January 2021

Cleavage of Structural Proteins during the Assembly of the Head of Bacteriophage T4

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7 January 2021

Identifiers

DOI

10.1038/3242

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:9809549%20AND%20SRC:MED&resulttype=core&format=json

6 December 2019

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