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Morpholinos and their peptide conjugates: Therapeutic promise and challenge for Duchenne muscular dystrophy

✍ Scribed by Hong M. Moulton; Jon D. Moulton

Book ID
116274960
Publisher
Elsevier Science
Year
2010
Tongue
English
Weight
463 KB
Volume
1798
Category
Article
ISSN
0005-2736

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## Abstract ## Background Antisense‐mediated exon skipping is a putative treatment for Duchenne muscular dystrophy (DMD). Using antisense oligonucleotides (AONs), the disrupted DMD reading frame is restored, allowing generation of partially functional dystrophin and conversion of a severe Duchenne