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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Bibliografiska uppgifter
Huvudupphovsmän: Disterer, Petra, Kryczka, Adrianna, Liu, Yuqi, Badi, Yusef E., Wong, Jessie J., Owen, James S., Khoo, Bernard
Materialtyp: Artigo
Språk:Inglês
Publicerad: Mary Ann Liebert, Inc. 2014
Ämnen:
Reviews
Länkar: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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