Multiple Exon Skipping in the Duchenne Muscular Dystrophy Hot Spots: Prospects and Challenges

Duchenne muscular dystrophy (DMD), a fatal X-linked recessive disorder, is caused mostly by frame-disrupting, out-of-frame deletions in the dystrophin (DMD) gene. Antisense oligonucleotide-mediated exon skipping is a promising therapy for DMD. Exon skipping aims to convert out-of-frame mRNA to in-fr...

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Kaydedildi:
Detaylı Bibliyografya
Yayımlandı:J Pers Med
Asıl Yazarlar: Echigoya, Yusuke, Lim, Kenji Rowel Q., Nakamura, Akinori, Yokota, Toshifumi
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: MDPI 2018
Konular:
Review
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC6313462/
https://ncbi.nlm.nih.gov/pubmed/30544634
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/jpm8040041
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