Journal article
Aminoglycoside‐induced Translational Read‐through in Disease: Overcoming Nonsense Mutations by Pharmacogenetic Therapy
Clinical pharmacology and therapeutics, Vol.81(1), pp.99-103
01/2007
DOI: 10.1038/sj.clpt.6100012
PMID: 17186006
Abstract
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
Details
- Title: Subtitle
- Aminoglycoside‐induced Translational Read‐through in Disease: Overcoming Nonsense Mutations by Pharmacogenetic Therapy
- Creators
- L V Zingman - Mayo ClinicS Park - Mayo ClinicT M Olson - Mayo ClinicA E Alekseev - Mayo ClinicA Terzic - Mayo Clinic
- Resource Type
- Journal article
- Publication Details
- Clinical pharmacology and therapeutics, Vol.81(1), pp.99-103
- DOI
- 10.1038/sj.clpt.6100012
- PMID
- 17186006
- ISSN
- 0009-9236
- eISSN
- 1532-6535
- Number of pages
- 5
- Language
- English
- Date published
- 01/2007
- Academic Unit
- Internal Medicine
- Record Identifier
- 9984359764102771
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