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Fixing Mutations With A Drug

Anonymous Coward writes | more than 6 years ago

Biotech 0

An anonymous reader writes "More than 1,800 different genetic disorders including Duchenne muscular dystrophy, cystic fibrosis, and spinal muscular dystrophy, can be caused by a particular type of mutation called a nonsense mutation. Forbes has a story (Stopping the Nonsense http://www.forbes.com/free_forbes/2007/0917/072.html?partner=yahoomag) on a drug to correct these mutations.

The magazine explains: "A gene is akin to a long sentence consisting of repeating patterns of those four letters (A, T, G, C); the sequence T-G-A is like a period at the end of the sentence. It tells the body where a gene ends. But sometimes the period is out of place — it has been wrongly inserted somewhere in the sentence. Thus the set of instructions to produce the muscle protein, dystrophin, isn't read in full; this causes the body to produce a defective form of dystrophin that is too short to do its job.""

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