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Aminoglycoside‐induced Translational Read‐through in Disease: Overcoming Nonsense Mutations by Pharmacogenetic Therapy
Author(s) -
Zingman LV,
Park S,
Olson TM,
Alekseev AE,
Terzic A
Publication year - 2007
Publication title -
clinical pharmacology and therapeutics
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.941
H-Index - 188
eISSN - 1532-6535
pISSN - 0009-9236
DOI - 10.1038/sj.clpt.6100012
Subject(s) - nonsense mutation , nonsense , pharmacogenetics , genetic enhancement , aminoglycoside , nonsense mediated decay , translation (biology) , medicine , phenotype , gene , disease , mutation , genetics , bioinformatics , biology , genotype , missense mutation , rna , antibiotics , rna splicing , messenger rna
A third of inherited diseases result from premature termination codon mutations. Aminoglycosides have emerged as vanguard pharmacogenetic agents in treating human genetic disorders due to their unique ability to suppress gene translation termination induced by nonsense mutations. In preclinical and pilot clinical studies, this therapeutic approach shows promise in phenotype correction by promoting otherwise defective protein synthesis. The challenge ahead is to maximize efficacy while preventing interaction with normal protein production and function. Clinical Pharmacology & Therapeutics (2007) 81 , 99–103. doi: 10.1038/sj.clpt.6100012