Quantitative Antisense Screening and Optimization for Exon 51 Skipping in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder, is caused by mutations in the dystrophin (DMD) gene. Exon skipping is a therapeutic approach that uses antisense oligonucleotides (AOs) to modulate splicing and restore the reading frame, leading to truncated, yet functional...

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書誌詳細
出版年:Mol Ther
主要な著者: Echigoya, Yusuke, Lim, Kenji Rowel Q., Trieu, Nhu, Bao, Bo, Miskew Nichols, Bailey, Vila, Maria Candida, Novak, James S., Hara, Yuko, Lee, Joshua, Touznik, Aleksander, Mamchaoui, Kamel, Aoki, Yoshitsugu, Takeda, Shin’ichi, Nagaraju, Kanneboyina, Mouly, Vincent, Maruyama, Rika, Duddy, William, Yokota, Toshifumi
フォーマット: Artigo
言語:Inglês
出版事項: American Society of Gene & Cell Therapy 2017
主題:
Original Article
オンライン･アクセス:https://ncbi.nlm.nih.gov/pmc/articles/PMC5675502/
https://ncbi.nlm.nih.gov/pubmed/28865998
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2017.07.014
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