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

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...

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Vydáno v:Mol Ther Nucleic Acids
Hlavní autoři: Watanabe, Naoki, Nagata, Tetsuya, Satou, Youhei, Masuda, Satoru, Saito, Takashi, Kitagawa, Hidetoshi, Komaki, Hirofumi, Takagaki, Kazuchika, Takeda, Shin’ichi
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2018
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6202794/
https://ncbi.nlm.nih.gov/pubmed/30388618
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtn.2018.09.017
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