기술동향
New Drug Bypasses Gene Mutations
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2008-11-14
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Technolog Rreview
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#Gene#Mutations #New Drug
출처 : Technolog Rreview
New Drug Bypasses Gene Mutations
A compound that helps cells produce normal proteins from wonky genes could have a broad impact on genetic diseases.
By Emily Singer
A novel drug that enables the production of normal proteins from mutated DNA might one day help people with a variety of genetic diseases. The drug has shown promise as a treatment for cystic fibrosis and muscular dystrophy, and it is now being tested in large, international clinical trials.
Most drugs alter the activity of proteins after they're manufactured, but the new drug intervenes in the cellular machinery that makes the proteins in the first place. Consequently, it could be effective against diseases where completely different proteins go awry. "It's a great breakthrough," says Robert Singer, a biologist at the Albert Einstein Medical School, in New York, who is not involved with the company that produces the drug.
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