SMRT compounds correct nonsense mutations in primary immunodeficiency and other genetic models.

Annals of the New York Academy of Sciences
Richard A Gatti

Abstract

Within less than 10 years after the realization of the double helix of DNA, the ability of aminoglycosides to influence the misreading or readthrough of premature termination codons was discovered. It took another three decades to clone and sequence disease genes and appreciate the similarity of mutation spectra for most inborn errors. Nonsense mutations once again have become the target of readthrough compounds. In this brief review, we trace the development in our laboratory of the next generation of readthrough agents, small molecule readthrough (SMRT) drug-like chemicals, and assays for comparing their in vitro activity. Possible mechanisms of action and potential clinical applications are considered.

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Citations

Jun 19, 2013·Molecular Therapy : the Journal of the American Society of Gene Therapy·Liutao DuRichard A Gatti
Nov 13, 2013·Proceedings of the National Academy of Sciences of the United States of America·Christiane KuschalKenneth H Kraemer
Feb 18, 2016·BioDrugs : Clinical Immunotherapeutics, Biopharmaceuticals and Gene Therapy·Kerstin Nagel-WolfrumUwe Wolfrum
Jul 14, 2016·Nucleic Acids Research·Alireza Baradaran-HeraviMichel Roberge
Dec 2, 2019·Journal of Molecular Medicine : Official Organ of the Gesellschaft Deutscher Naturforscher Und Ärzte·Amnon WittensteinRina Rosin-Arbesfeld

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Aminoglycosides (ASM)

Aminoglycoside is a medicinal and bacteriologic category of traditional Gram-negative antibacterial medications that inhibit protein synthesis and contain as a portion of the molecule an amino-modified glycoside. Discover the latest research on aminoglycoside here.

Aminoglycosides

Aminoglycoside is a medicinal and bacteriologic category of traditional Gram-negative antibacterial medications that inhibit protein synthesis and contain as a portion of the molecule an amino-modified glycoside. Discover the latest research on aminoglycoside here.