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RNA-modulating therapeutics: personalized exon skipping for duchenne muscular dystrophy

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RNA-modulating therapeutics: personalized exon skipping for duchenne muscular dystrophy

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_20239163

RNA-modulating therapeutics: personalized exon skipping for duchenne muscular dystrophy

About this item

Full title

RNA-modulating therapeutics: personalized exon skipping for duchenne muscular dystrophy

Author / Creator

Van Deutekom, Judith , de Kimpe, Sjef , Ekhart, Peter , van den Akker, Hanneke , Sitsen, Ad , Janson, Anneke , de Winter, Christa , Aartsma-Rus, Annemieke , Ommen, Gert-Jan van , Verschuuren, Jan , Goemans, Nathalie , Tulinius, Mar and Platenburg, Gerard

Publisher

Mary Ann Liebert, Inc

Journal title

Human gene therapy, 2008-10, Vol.19 (10), p.1062-1062

Language

English

Formats

Articles

Publication information

Publisher

Mary Ann Liebert, Inc

Subjects

More information

Scope and Contents

Contents

Duchenne muscular dystrophy (DMD) patients suffer from a progressive, severe muscle-wasting disease due to frame-disrupting mutations in the DMD gene and a complete loss of functional dystrophin. Antisense oligonucleotide compounds (AONs) have recently shown therapeutic promise for DMD patients. By inducing specific exon skipping during mRNA splici...

Alternative Titles

Full title

RNA-modulating therapeutics: personalized exon skipping for duchenne muscular dystrophy

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Author / Creator

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Primary Identifiers

Record Identifier

TN_cdi_proquest_miscellaneous_20239163

Permalink

https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_20239163

Other Identifiers

ISSN

1043-0342

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