Development of Therapeutic Splice-Switching Oligonucleotides

Synthetic splice-switching oligonucleotides (SSOs) target nuclear pre-mRNA molecules to change exon splicing and generate an alternative protein isoform. Clinical trials with two competitive SSO drugs are underway to treat Duchenne muscular dystrophy (DMD). Beyond DMD, many additional therapeutic ap...

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Detalhes bibliográficos
Main Authors: Disterer, Petra, Kryczka, Adrianna, Liu, Yuqi, Badi, Yusef E., Wong, Jessie J., Owen, James S., Khoo, Bernard
Formato: Artigo
Idioma:Inglês
Publicado em: Mary Ann Liebert, Inc. 2014
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4098814/
https://ncbi.nlm.nih.gov/pubmed/24826963
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.234
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