The minor gentamicin complex component, X2, is a potent premature stop codon readthrough molecule with therapeutic potential.

Nonsense mutations, resulting in a premature stop codon in the open reading frame of mRNAs are responsible for thousands of inherited diseases. Readthrough of premature stop codons by small molecule drugs has emerged as a promising therapeutic approach to treat disorders resulting from premature ter...

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Bibliographic Details
Main Authors: Westley J Friesen, Briana Johnson, Jairo Sierra, Jin Zhuo, Priya Vazirani, Xiaojiao Xue, Yuki Tomizawa, Ramil Baiazitov, Christie Morrill, Hongyu Ren, Suresh Babu, Young-Choon Moon, Art Branstrom, Anna Mollin, Jean Hedrick, Josephine Sheedy, Gary Elfring, Marla Weetall, Joseph M Colacino, Ellen M Welch, Stuart W Peltz
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2018-01-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC6201930?pdf=render