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Biochemistry, Biophysics, and Structural Biology Commons

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Physiology

2007

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Full-Text Articles in Biochemistry, Biophysics, and Structural Biology

Data From: Ptc124 Targets Genetic Disorders Caused By Nonsense Mutations, Allan Jacobson, Phyllis Spatrick, Feng He May 2007

Data From: Ptc124 Targets Genetic Disorders Caused By Nonsense Mutations, Allan Jacobson, Phyllis Spatrick, Feng He

MaPS Research Data

Manuscript abstract: Nonsense mutations promote premature translational termination and cause anywhere from 5-70% of the individual cases of most inherited diseases. Studies on nonsense-mediated cystic fibrosis have indicated that boosting specific protein synthesis from less than 1% to as little as 5% of normal levels may greatly reduce the severity or eliminate the principal manifestations of disease. To address the need for a drug capable of suppressing premature termination, we identified PTC124-a new chemical entity that selectively induces ribosomal readthrough of premature but not normal termination codons. PTC124 activity, optimized using nonsense-containing reporters, promoted dystrophin production in primary muscle cells ...