Targeting Translation Termination Machinery with Antisense Oligonucleotides for Diseases Caused by Nonsense Mutations

Efforts to develop treatments for diseases caused by nonsense mutations have focused on identification of small molecules that promote translational read-through of messenger RNAs (mRNAs) harboring nonsense stop codons to produce full-length proteins. However, to date, no small molecule read-through...

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Dettagli Bibliografici
Pubblicato in:Nucleic Acid Ther
Autori principali: Huang, Lulu, Aghajan, Mariam, Quesenberry, Tianna, Low, Audrey, Murray, Susan F., Monia, Brett P., Guo, Shuling
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc., publishers 2019
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6686700/
https://ncbi.nlm.nih.gov/pubmed/31070517
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/nat.2019.0779
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