Targeting nuclear RNA for in vivo correction of myotonic dystrophy
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
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Publisher
London: Nature Publishing Group UK
Journal title
Language
English
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Publication information
Publisher
London: Nature Publishing Group UK
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Scope and Contents
Contents
Nuclear-retained transcripts containing expanded repeats are shown to be sensitive to antisense silencing, and in a transgenic mouse model of myotonic dystrophy type 1, systemic administration of ASOs causes a rapid knockdown of the toxic RNA in skeletal muscle, correcting some hallmark features of the disease.
A muscular dystrophy rescued by an...
Alternative Titles
Full title
Targeting nuclear RNA for in vivo correction of myotonic dystrophy
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Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4221572
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4221572
Other Identifiers
ISSN
0028-0836
E-ISSN
1476-4687
DOI
10.1038/nature11362
