Rational design of antisense oligomers to induce dystrophin exon skipping. (Q33713852)

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23 January 2020

title

Rational design of antisense oligomers to induce dystrophin exon skipping (English)

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23 January 2020

1

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23 January 2020

Sue Fletcher

5

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23 January 2020

4

Francesco Muntoni

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23 January 2020

author name string

Abbie M Adams

2

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23 January 2020

Penny L Meloni

3

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23 January 2020

Steve D Wilton

6

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23 January 2020

publication date

17 March 2009

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23 January 2020

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23 January 2020

volume

17

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23 January 2020

issue

8

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23 January 2020

page(s)

1418-1426

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Splicing regulation: from a parts list of regulatory elements to an integrated splicing code

23 January 2020

cites work

1 reference

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Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries

21 June 2018

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The influence of antisense oligonucleotide length on dystrophin exon skipping.

21 June 2018

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Altered splicing in prelamin A-associated premature aging phenotypes

21 June 2018

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An increased specificity score matrix for the prediction of SF2/ASF-specific exonic splicing enhancers

21 June 2018

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Comparative analysis identifies exonic splicing regulatory sequences--The complex definition of enhancers and silencers

21 June 2018

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Induction of revertant fibres in the mdx mouse using antisense oligonucleotides

21 June 2018

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Functional analysis of 114 exon-internal AONs for targeted DMD exon skipping: indication for steric hindrance of SR protein binding sites

21 June 2018

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The architecture of pre-mRNAs affects mechanisms of splice-site pairing.

21 June 2018

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Experience and strategy for the molecular testing of Duchenne muscular dystrophy

21 June 2018

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Rules of engagement: co-transcriptional recruitment of pre-mRNA processing factors

21 June 2018

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

21 June 2018

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Towards a therapeutic inhibition of dystrophin exon 23 splicing in mdx mouse muscle induced by antisense oligoribonucleotides (splicomers): target sequence optimisation using oligonucleotide arrays

21 June 2018

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Transcriptome and genome conservation of alternative splicing events in humans and mice.

21 June 2018

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How prevalent is functional alternative splicing in the human genome?

21 June 2018

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The spliceosome: the most complex macromolecular machine in the cell?

21 June 2018

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Pre-mRNA splicing: awash in a sea of proteins

21 June 2018

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ESEfinder: A web resource to identify exonic splicing enhancers

21 June 2018

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Mechanisms of alternative pre-messenger RNA splicing

21 June 2018

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Protein composition of human prespliceosomes isolated by a tobramycin affinity-selection method

21 June 2018

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Comprehensive proteomic analysis of the human spliceosome

21 June 2018

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Predictive identification of exonic splicing enhancers in human genes

21 June 2018

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Muscular dystrophy into the new millennium

21 June 2018

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Listening to silence and understanding nonsense: exonic mutations that affect splicing

21 June 2018

1 reference

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Function and genetics of dystrophin and dystrophin-related proteins in muscle

21 June 2018

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In situ transcription and splicing in the Balbiani ring 3 gene

21 June 2018

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Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse

21 June 2018

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Induction of exon skipping of the dystrophin transcript in lymphoblastoid cells by transfecting an antisense oligodeoxynucleotide complementary to an exon recognition sequence

21 June 2018

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Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides

21 June 2018

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Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy

21 June 2018

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Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript.

21 June 2018

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Antisense oligonucleotide-induced exon skipping restores dystrophin expression in vitro in a canine model of DMD.

30 October 2018

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Targeted exon skipping in transgenic hDMD mice: A model for direct preclinical screening of human-specific antisense oligonucleotides

30 October 2018

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Maximum entropy modeling of short sequence motifs with applications to RNA splicing signals

30 October 2018

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Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene.

30 October 2018

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Targeted exon skipping as a potential gene correction therapy for Duchenne muscular dystrophy.

30 October 2018

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DMD pseudoexon mutations: splicing efficiency, phenotype, and potential therapy

30 October 2018

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12 December 2020

Deletion of exon 16 of the dystrophin gene is not associated with disease

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12 December 2020

The muscular dystrophies

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12 December 2020

By-passing the nonsense mutation in the 4 CV mouse model of muscular dystrophy by induced exon skipping

1 reference

12 December 2020

Splicing mosaic of the myophosphorylase gene due to a silent mutation in McArdle disease

1 reference

12 December 2020

Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy

1 reference

12 December 2020

Local dystrophin restoration with antisense oligonucleotide PRO051

1 reference

12 December 2020

Identifiers

DOI

10.1038/MT.2009.49

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:19293776 AND SRC:MED&resulttype=core&format=json

23 January 2020

1 reference

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23 January 2020

PubMed publication ID

19293776

1 reference