(Q45881319)

17 December 2017

title

Gene therapy: the 'pro-sense' approach to Duchenne muscular dystrophy (English)

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17 December 2017

main subject

gene therapy

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author name string

Judith C T van Deutekom

series ordinal

1

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17 December 2017

publication date

1 May 2005

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17 December 2017

European Journal of Human Genetics

published in

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17 December 2017

volume

13

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17 December 2017

issue

5

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17 December 2017

page(s)

518-519

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17 December 2017

https://scigraph.springernature.com/pub.10.1038/sj.ejhg.5201381

exact match

0 references

cites work

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

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

Advances in Duchenne muscular dystrophy gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

An explanation for the phenotypic differences between patients bearing partial deletions of the DMD locus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

Double-target antisense U7 snRNAs promote efficient skipping of an aberrant exon in three human beta-thalassemic mutations

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.EJHG.5201381

21 January 2018

Identifiers

DOI

10.1038/SJ.EJHG.5201381

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17 December 2017

Dimensions Publication ID

1031091877

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1 reference

17 December 2017