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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Hlavní autoři: Disterer, Petra, Kryczka, Adrianna, Liu, Yuqi, Badi, Yusef E., Wong, Jessie J., Owen, James S., Khoo, Bernard
Médium: Artigo
Jazyk:Inglês
Vydáno: Mary Ann Liebert, Inc. 2014
Témata:
Reviews
On-line přístup: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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