Exon Skipping Using Antisense Oligonucleotides for Laminin-Alpha2-Deficient Muscular Dystrophy
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Publisher
Springer New York
Link
http://link.springer.com/content/pdf/10.1007/978-1-4939-8651-4_36
Reference10 articles.
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2. Wood MJ (2010) Toward an oligonucleotide therapy for Duchenne muscular dystrophy: a complex development challenge. Sci Transl Med 2:25ps15
3. Aoki Y, Nakamura A, Yokota T et al (2010) In-frame dystrophin following exon 51-skipping improves muscle pathology and function in the exon 52-deficient mdx mouse. Mol Ther 18:1995–2005
4. Aoki Y, Yokota T, Nagata T et al (2012) Bodywide skipping of exons 45–55 in dystrophic mdx52 mice by systemic antisense delivery. Proc Natl Acad Sci U S A 109:13763–13768
5. Yokota T, Lu QL, Partridge T et al (2009) Efficacy of systemic morpholino exon-skipping in Duchenne dystrophy dogs. Ann Neurol 65:667–676
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