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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Bibliografiske detaljer
Udgivet i:Nucleic Acid Ther
Main Authors: Huang, Lulu, Aghajan, Mariam, Quesenberry, Tianna, Low, Audrey, Murray, Susan F., Monia, Brett P., Guo, Shuling
Format: Artigo
Sprog:Inglês
Udgivet: Mary Ann Liebert, Inc., publishers 2019
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Original Articles
Online adgang: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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