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Treatment for progeria?

Two common drugs have reversed the effects of progeria in mice--and they have few side-effects, so may one day be used in children with the disease. Progeria accelerates from early childhood and is usually fatal before puberty. There is currently no cure. The disease is caused by gene mutations that disrupt production of the protein prelamin A, found inside the nuclei of cells. The damaged prelamin A binds to molecular fragments in the body called farnesyls, which then bind to the nuclear membrane, causing the build-up of protein that underlies the disease.

Statins and bisphosphonates are known to reduce farnesyl levels. This led Carlos Lopez-Otin to speculate that they may be able to reverse progeria. The team gave a mixture of the two drugs to progerid mice and found that this reduced ageing symptoms and the mice lived longer than controls. Researchers are now seeking permission for research on humans.

Nature Medicine, DOI:10.1038/nm1786

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Title Annotation:Single Suture
Publication:CME: Your SA Journal of CPD
Article Type:Brief article
Geographic Code:6SOUT
Date:Sep 1, 2008
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