Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot

Duchenne muscular dystrophy (DMD) is a severe muscular disorder. It was reported that multiple exon skipping (MES), targeting exon 45–55 of the DMD gene, might improve patients’ symptoms because patients who have a genomic deletion of all these exons showed very mild symptoms. Thus, exon 45–55 skipp...

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Veröffentlicht in:Int J Mol Sci
Hauptverfasser: Suzuki, Hitoshi, Aoki, Yoshitsugu, Kameyama, Toshiki, Saito, Takashi, Masuda, Satoru, Tanihata, Jun, Nagata, Tetsuya, Mayeda, Akila, Takeda, Shin’ichi, Tsukahara, Toshifumi
Format: Artigo
Sprache:Inglês
Veröffentlicht: MDPI 2016
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Article
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC5085753/
https://ncbi.nlm.nih.gov/pubmed/27754374
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.3390/ijms17101722
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