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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Bibliografiske detaljer
Udgivet i:Mol Ther
Main Authors: 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
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2017
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Original Article
Online adgang: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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