Abstract | INTRODUCTION: AREAS COVERED: In this review, we focus on the current progress and challenges of exon-skipping and splice modulation therapies. In addition, we discuss the recent failure of the Phase III clinical trials of exon 51 skipping ( drisapersen) for DMD. EXPERT OPINION:
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Authors | Aleksander Touznik, Joshua J A Lee, Toshifumi Yokota |
Journal | Expert opinion on biological therapy
(Expert Opin Biol Ther)
Vol. 14
Issue 6
Pg. 809-19
(Jun 2014)
ISSN: 1744-7682 [Electronic] England |
PMID | 24620745
(Publication Type: Journal Article, Research Support, Non-U.S. Gov't, Review)
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Chemical References |
- Oligonucleotides, Antisense
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Topics |
- Animals
- Exons
- Gene Expression Regulation
- Genetic Therapy
(methods)
- Humans
- Muscular Atrophy, Spinal
(genetics, physiopathology, therapy)
- Muscular Dystrophies, Limb-Girdle
(genetics, physiopathology, therapy)
- Muscular Dystrophy, Duchenne
(genetics, physiopathology, therapy)
- Oligonucleotides, Antisense
(therapeutic use)
- RNA Splicing
- Treatment Outcome
- Walker-Warburg Syndrome
(genetics, physiopathology, therapy)
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