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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Detalhes bibliográficos
Publicado no: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
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2017
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Original Article
Acesso em linha: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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