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Editorial
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Volume 358:190-192 January 10, 2008 Number 2
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Efficacy of Sirolimus in Treating Tuberous Sclerosis and Lymphangioleiomyomatosis
Elahna Paul, M.D., Ph.D., and Elizabeth Thiele, M.D., Ph.D.

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 by Bissler, J. J.
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Owing to their immunosuppressive and antiproliferative effects, sirolimus (also called rapamycin) and related drugs are being evaluated as part of many transplant immunosuppresion regimens, as well as for a plethora of medical conditions such as type 1 diabetes, macular degeneration, coronary artery disease, and metastatic or refractory cancers of the breast, prostate, lung, and liver, to name but a few.

The effects of sirolimus are mediated by its inhibition of the curiously named cytoplasmic protein mammalian target of rapamycin (mTOR), a ubiquitous serine–threonine kinase that is intimately involved in the regulation of protein synthesis, cell growth, cytoskeletal organization, and other . . . [Full Text of this Article]


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From the Division of Pediatric Nephrology (E.P.) and the Departments of Neurology and Pediatrics (E.T.), Massachusetts General Hospital, Boston.


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