NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

• Main Authors: Naoki Watanabe (Author), Tetsuya Nagata (Author), Youhei Satou (Author), Satoru Masuda (Author), Takashi Saito (Author), Hidetoshi Kitagawa (Author), Hirofumi Komaki (Author), Kazuchika Takagaki (Author), Shin'ichi Takeda (Author)
• Format: Book
• Published: Elsevier, 2018-12-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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 restor...