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Comment
. 2013;11(6):e1001458.
doi: 10.1371/journal.pbio.1001458. Epub 2013 Jun 25.

A read-through drug put through its paces

Roland G Roberts  1
Affiliations
Comment

A read-through drug put through its paces

Roland G Roberts. PLoS Biol. 2013.
No abstract available

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Conflict of interest statement

The author has declared that no competing interests exist.

Figures

Figure 1
Figure 1. When good assays turn bad.
a) A ribosome (dark purple) runs along a normal transcript (wiggly orange/green line), translating it into protein (orange circle – upstream region; green sun – luciferase), which then emits light in the assay. b) A nonsense mutation (red hexagon) stops most of the ribosomes in the middle of the transcript, producing a truncated, non-luminescent protein (orange circle), with very few ribosomes (pale purple) reading through to give a dim luciferase light signal in the assay. The drug (blue triangle) has no effect. c) The dream scenario—a drug interacts with the ribosome, causing it to read through, generating plenty of luciferase and light. d) The potential explanation for the discovery of PTC124—the drug instead interacts with the luciferase molecules, stabilizing them (large green sun) and giving a spurious light signal.
See this image and copyright information in PMC

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