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Study of antisense oligonucleotide phosphorothioates containing segments of oligodeoxynucleotides and 2′-o- methyloligoribonucleotides

✍ Scribed by Valeri Metelev; Julianna Lisziewicz; Sudhir Agrawal


Book ID
108477024
Publisher
Elsevier Science
Year
1994
Tongue
English
Weight
419 KB
Volume
4
Category
Article
ISSN
0960-894X

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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