Ontology highlight
ABSTRACT:
SUBMITTER: Watanabe N
PROVIDER: S-EPMC6202794 | biostudies-literature | 2018 Dec
REPOSITORIES: biostudies-literature
Watanabe Naoki N Nagata Tetsuya T Satou Youhei Y Masuda Satoru S Saito Takashi T Kitagawa Hidetoshi H Komaki Hirofumi H Takagaki Kazuchika K Takeda Shin'ichi S
Molecular therapy. Nucleic acids 20180927
Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein. Exon skipping mediated by antisense oligonucleotides has recently emerged as an effective approach for the restoration of dystrophin, and skipping of exon 51 of DMD has received accelerated approval. Identifying antisense sequences that can provide the highest possible skipping efficiency is crucial for future c ...[more]