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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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Detalles Bibliográficos
Autores principales: Huang, Lulu, Aghajan, Mariam, Quesenberry, Tianna, Low, Audrey, Murray, Susan F., Monia, Brett P., Guo, Shuling
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2019
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6686700/
https://www.ncbi.nlm.nih.gov/pubmed/31070517
http://dx.doi.org/10.1089/nat.2019.0779