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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Bibliographic Details
Main Authors: Disterer, Petra, Kryczka, Adrianna, Liu, Yuqi, Badi, Yusef E., Wong, Jessie J., Owen, James S., Khoo, Bernard
Format: Artigo
Language:Inglês
Published: Mary Ann Liebert, Inc. 2014
Subjects:
Reviews
Online Access: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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