Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin...
Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice
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Publisher
United States: Elsevier Inc
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Language
English
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Publisher
United States: Elsevier Inc
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Contents
Antisense oligonucleotide (AO)–mediated splice correction therapy for Duchenne muscular dystrophy has shown huge promise from recent phase 2b clinical trials, however high doses and costs are required and targeted delivery can lower both of these. We have previously demonstrated the feasibility of targeted delivery of AOs by conjugating a chimeric...
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Full title
Effective Dystrophin Restoration by a Novel Muscle-Homing Peptide–Morpholino Conjugate in Dystrophin-Deficient mdx Mice
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TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4088999
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https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_4088999
Other Identifiers
ISSN
1525-0016
E-ISSN
1525-0024
DOI
10.1038/mt.2014.63
