Article
Endogenous Multiple Exon Skipping and Back-Splicing at the DMD Mutation Hotspot.
International journal of molecular sciences - 13 Oct 2016
Suzuki Hitoshi, Aoki Yoshitsugu, Kameyama Toshiki, Saito Takashi, Masuda Satoru, Tanihata Jun, Nagata Tetsuya, Mayeda Akila, Takeda Shin'ichi, Tsukahara Toshifumi
Abstract excerpt
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 skipping treatments for DMD have been proposed as a potential clinical cure. Herein, we detected the expression of...
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