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Biological Chemistry

Rapamycin Diversifies To Fight Early Aging

by Stu Borman
July 4, 2011 | A version of this story appeared in Volume 89, Issue 27

The immunosuppressant drug rapamycin typically given to organ transplant patients appears to have a new use: treating progeria. Progeria is a rare genetic condition in which young children show signs of aging and die prematurely. There are no proven treatments. The disease is caused by a single-nucleotide gene substitution that leads to production of a mutant protein called progerin, which causes cell senescence. Rapamycin had been shown previously to help prevent aging of cells and organisms. Francis S. Collins of the National Human Genome Research Institute, Dimitri Krainc of Massachusetts General Hospital and Harvard Medical School, and coworkers now report that rapamycin delays premature aging and helps prevent formation of progerin aggregates in cells with the progeria mutation (Sci. Transl. Med., DOI: 10.1126/scitranslmed.3002346). From a mechanistic standpoint, rapamycin activates a lysosomal pathway that degrades accumulated progerin. According to the researchers, the findings could lead to a clinical trial of the rapamycin analog everolimus in children with progeria “and may offer insights into normal aging as well.”


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