In vivo correction of a Menkes disease model using antisense oligonucleotides (Q36499173)

14 August 2017

In vivo correction of a Menkes disease model using antisense oligonucleotides (English)

1 reference

14 August 2017

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Erik C Madsen

1

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14 August 2017

Paul A Morcos

series ordinal

2

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14 August 2017

Bryce A Mendelsohn

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3

1 reference

14 August 2017

Jonathan D Gitlin

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4

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14 August 2017

language of work or name

English

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publication date

3 March 2008

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Proceedings of the National Academy of Sciences of the United States of America

14 August 2017

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14 August 2017

105

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14 August 2017

10

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14 August 2017

3909-3914

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Propionic and methylmalonic acidemia: antisense therapeutics for intronic variations causing aberrantly spliced messenger RNA

14 August 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Splicing in disease: disruption of the splicing code and the decoding machinery

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo.

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Function and regulation of human copper-transporting ATPases

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Modulating the expression of disease genes with RNA-based therapy.

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Essential role of lysyl oxidases in notochord development

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Correction of prototypic ATM splicing mutations and aberrant ATM function with antisense morpholino oligonucleotides

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Functional copper transport explains neurologic sparing in occipital horn syndrome

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Structural basis for polypyrimidine tract recognition by the essential pre-mRNA splicing factor U2AF65.

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Copper-replacement treatment for symptomatic Menkes disease: ethical considerations

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Variation in sequence and organization of splicing regulatory elements in vertebrate genes

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Correction of aberrant FGFR1 alternative RNA splicing through targeting of intronic regulatory elements

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Protein tolerance to random amino acid change

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Zebrafish: a genetic model for vertebrate organogenesis and human disorders

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Function and regulation of the mammalian copper-transporting ATPases: insights from biochemical and cell biological approaches

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Metabolic and molecular bases of Menkes disease and occipital horn syndrome

30 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=2268804

Correction of a mouse model of Menkes disease by the human Menkes gene.

30 September 2017

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0710865105

Atp7a determines a hierarchy of copper metabolism essential for notochord development

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0710865105

Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle.

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0710865105

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0710865105

Translational control of dosage compensation in Drosophila by Sex-lethal: cooperative silencing via the 5' and 3' UTRs of msl-2 mRNA is independent of the poly(A) tail

21 January 2018

1 reference

https://api.crossref.org/works/10.1073%2FPNAS.0710865105

21 January 2018

Identifiers

DOI

10.1073/PNAS.0710865105

1 reference

release_yb64uhg3qjcovbxle6m2f372cy

14 August 2017

0 references

1 reference

Fatcat

https://api.fatcat.wiki/v0/release/yb64uhg3qjcovbxle6m2f372cy

mapped directly with Wikidata item

24 November 2022

based on heuristic

1 reference

14 August 2017

1 reference